Company Presentations

Partnering Opportunities Abound!

See the 70+ companies who presented at the 2017 Cell & Gene Meeting on the Mesa Partnering Forum and were also available for one-on-one partnering meetings at the conference.

2017 Company Presenters

4DMT_70Abeona
Acucela_80Adaptimmune_95
Adverum_80AGTC
AGT_40Asterias
Athersys_45Audentes_90
AvroBio_80Bellicum_small-square_102x73
Benitec_80BioTime_85
blue bird bioBlueRock_90
B-Mogen_80Bone-Therapeutics_95
Caladrius_80Capricor
CARsgen_80Cell-Medica_90
CellSeed_85CDI_new
Cesca_75CRISPR_40
CynataDiscGenics_100
Fate_50Fibrocell_90
GammaDelta_95Healios_85
Histogenics2Homology
IntelliajCyte_55
JuventasLogicBio_80
Lysogene_85MaxCyte
Medeor_75MEDIPOST_70
Mesoblast_100MolMed2
NYSCFOrbsen_100
Orchard-Therapeutics_70Oxford-BioMedica
Precision-Biosciences_90Promethera_90
REGENXBIOReNeuron_80
Renova_70RoosterBio_95
Sangamo_80SCM-Lifescience
Semma_80Sentien_65
Sigilon_65StemBioSys
Synpromics_80Terumo-BCT
TxCell_70uniQure
UniversalCells_75Vericel
VeriGraft_90ViaCyte
Vivet_80WindMil_90

4DMT_140x65

4D MOLECULAR THERAPEUTICS
David Kirn, M.D., Co-Founder and CEO
Oct. 5 | 4:00pm | La Jolla Ballroom 2
Emeryville, CA
(Private)
The mission of 4D Molecular Therapeutics (4DMT) is to design and develop transformative gene therapy products using the company’s proprietary technology to create novel adeno-associated virus (AAV) variants to be utilized as treatments for serious unmet medical conditions. 4DMT’s products are unlocking the full potential of gene therapy to treat, and potentially cure, genetic diseases. A large number of patients and diseases, who previously were not treatable by gene therapy, will be addressable by 4DMT products. www.4dmoleculartherapeutics.com

Abeona_140x65

ABEONA THERAPEUTICS
Tim Miller, Ph.D., President and CEO
Oct. 5 | 2:30pm | La Jolla Ballroom 1
Dallas, TX
(NASDAQ: ABEO)
Abeona Therapeutics is a clinical-stage biopharmaceutical company developing gene therapies for life-threatening rare genetic diseases. Abeona’s lead programs include ABO-102 (AAV-SGSH), an adeno-associated virus (AAV)-based gene therapy for Sanfilippo syndrome type A (MPS IIIA) and EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB). Abeona is also developing ABO-101 (AAV-NAGLU) for Sanfilippo syndrome type B (MPS IIIB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JNCL), ABO-202 (AAV-CLN1) for treatment of infantile Batten disease (INCL), EB-201 for epidermolysis bullosa (EB), ABO-301 (AAV-FANCC) for Fanconi anemia (FA) disorder and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy for rare blood diseases. In addition, Abeona has a proprietary vector platform, AIM™, for next generation product candidates. www.abeonatherapeutics.com

Acucela_140x65

ACUCELA
Lukas Scheibler, Ph.D., EVP, Research and Development
Oct. 4 | 5:45pm | La Jolla Ballroom 2
Seattle, WA
(TSE: 4589)
Acucela is a Seattle-based, Kubota Pharmaceutical Group company committed to translating innovation into a diverse portfolio of drugs and devices to preserve and restore vision for millions of people worldwide. Acucela currently develops a retina focused pipeline. It includes technologies for age-related macular degeneration (AMD), diabetic retinopathy and the orphan indications, retinitis pigmentosa (RP) and Stargardt disease (STGD). The RP program is an optogenetic adeno-associated virus (AAV) gene therapy vector that targets human rhodopsin to the ON bipolar cells of the RP patient’s retina to restore visual signal. www.acucela.com

Adaptimmune_165x65

ADAPTIMMUNE
Kimberly Freeman, VP, Commercial Planning
Oct. 4 | 2:45pm | La Jolla Ballroom 1
Oxford, UK
(NASDAQ: ADAP)
Adaptimmune, a leader in T-cell therapy to treat cancer, has multiple trials ongoing in both solid tumors and hematologic cancer types, and in cancers where survival rates for patients can be very limited. The company’s T-cell therapies have already shown preliminary evidence of tumor reduction in patients and also show a promising risk/benefit profile. Developed over the last 15 years, Adaptimmune’s proprietary SPEAR® (Specific Peptide Engineered Affinity Receptor) T-cell engineering platform has generated a strong pipeline of affinity enhanced T-cell therapies. The company uses these therapies to harness the body’s own immune system to find and destroy diseased cells. Adaptimmune can develop T-cell therapies for a broad spectrum of cancer types and patients, and have multiple T-cell therapies in development and/or in clinical trials. The company’s SPEAR TCR therapies offer promise to patients that often have no other options. Adaptimmune is working hard to make that promise a reality. www.adaptimmune.com

Adverum_150x65

ADVERUM BIOTECHNOLOGIES
Amber Salzman, Ph.D., President and CEO
Oct. 5 | 2:15pm | La Jolla Ballroom 2
Menlo Park, CA
(NASDAQ: ADVM)
Adverum Biotechnologies is a gene therapy company committed to discovering and developing novel medicines that can offer life-changing benefits to patients living with rare diseases or diseases of the eye who currently have limited or burdensome treatment options. Adverum has a robust pipeline and is leveraging its next generation adeno-associated virus (AAV)-based directed evolution platform to generate product candidates designed to provide durable efficacy by inducing sustained expression of a therapeutic protein. The company’s focus on the patient is supported by clinical development expertise and core capabilities in vector optimization, process development, manufacturing and assay development. www.adverum.com

AGTC_100x65

AGTC
Sue Washer, President and CEO
Oct. 4 | 11:15am | La Jolla Ballroom 1
Alachua, FL
(NASDAQ: AGTC)
AGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients. AGTC’s lead product candidates are designed to treat inherited orphan diseases of the eye that significantly affect visual function and currently lack effective medical treatments. AGTC’s product pipeline includes ophthalmology programs in X-linked retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP), achromatopsia, wet AMD, and an optogenetics program with Bionic Sight. AGTC’s non-ophthalmology programs include its adrenoleukodystrophy program and its otology program, and the company expects to advance several otology product candidates into clinical development in the next few years. Each of AGTC’s XLRS, XLRP and adrenoleukodystrophy programs is partnered with Biogen. AGTC employs a highly-targeted approach to selecting and designing its product candidates, choosing to develop therapies for indications having high unmet medical need that it believes are clinically feasible and present commercial opportunities. www.agtc.com

AGT_65x65

AMERICAN GENE TECHNOLOGIES
David Pauza, Ph.D., Chief Science Officer
Oct. 5 | 10:45am | La Jolla Ballroom 2
Rockville, MD
(Private)
American Gene Technologies (AGT), is an emerging genetic medicine company with a proprietary lentiviral platform capable of broad applications including large and orphan indications, immuno-oncology and monogenic disorders. AGT will enter the clinic in 2017 with a Phase I clinical trial to evaluate AGT103 as a functional cure for HIV. Pre-IND applications for phenylketonuria (PKU) and hepatocellular carcinoma (HCC) will follow. These therapies demonstrate the breadth of AGT’s unique lentiviral platform, including such innovations as a Transient Vector™ for temporary (non-integrating) lentiviral expression and an ImmunoTox™ vector for stimulating anti-tumor immune-response in immuno-oncology applications. AGT continues to strengthen its patent pipeline and IP protection that now contains 10 patent families providing multi-factored “picket fence” protection around key developments and technological innovations. AGT’s proprietary platform innovations enable rapid therapeutic discovery at a fraction of the cost of other methods and platforms. www.americangene.com

Asterias_150x65

ASTERIAS BIOTHERAPEUTICS
Katy Spink, Ph.D., EVP and Chief Operating Officer
Oct. 5 | 5:00pm | La Jolla Ballroom 1
Fremont, CA
(NASDAQ: AST)
Asterias Biotherapeutics is a leading biotechnology company in the emerging field of regenerative medicine. The company’s proprietary cell therapy programs are based on its immunotherapy and pluripotent stem cell platform technologies. Asterias is presently focused on advancing three clinical-stage programs which have the potential to address areas of very high unmet medical need in the fields of oncology and neurology. www.asteriasbiotherapeutics.com

Athersys_60x65

ATHERSYS
Robert Mays, Ph.D., VP of Regenerative Medicine and Head of Neuroscience
Oct. 5 | 4:30pm | La Jolla Ballroom 1
Cleveland, OH
(NASDAQ: ATHX)
Athersys is a clinical-stage biotechnology company developing novel and proprietary best-in-class therapies designed to extend and enhance the quality of human life. The company’s focus is on the treatment of medical conditions where there is significant unmet clinical need, and Athersys is particularly focused on developing therapies in the regenerative medicine area. Athersys is developing MultiStem®, a patented, adult-derived off-the-shelf stem cell product platform for multiple disease indications in the areas of neurological, cardiovascular and inflammatory and immune disease areas, as well as other indications where there is unmet medical need. Athersys currently has six clinical-stage programs – including a pending Phase III study in ischemic stroke, an ongoing Phase II clinical study for the treatment of damage from acute myocardial infarction, an ongoing clinical study in acute respiratory distress syndrome and several others. The company also has a portfolio of preclinical programs advancing toward clinical-stage development. www.athersys.com

Audentes_150x65

AUDENTES THERAPEUTICS
Matt Patterson, President and CEO
Oct. 4 | 11:30am | La Jolla Ballroom 1
San Francisco, CA
(NASDAQ: BOLD)
Audentes Therapeutics is a biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases. The company has four product candidates in development, AT132 for the treatment of X-linked myotubular myopathy (XLMTM), AT342 for the treatment of Crigler-Najjar syndrome, AT982 for the treatment of Pompe disease, and AT307 for the treatment of the CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia (CASQ2-CPVT). Audentes is a focused, experienced and passionate team committed to forging strong, global relationships with the patient, research and medical communities. www.audentestx.com

AvroBio_130x65

AVROBIO
Geoff MacKay, President and CEO
Oct. 5 | 11:00am | La Jolla Ballroom 2
Cambridge, MA
(Private)
AVROBIO, a leader in lentiviral-based gene therapies, is a clinical-stage company developing disruptive therapies that have the potential to transform patients’ lives in a single dose. The company is focused on the development of its Phase I programs in Fabry disease and acute myeloid leukemia (AML), while actively building a pipeline of therapies to treat Gaucher disease and other rare diseases.
www.avrobio.com

Bellicum_140x65

BELLICUM PHARMACEUTICALS
Gregory Naeve, Ph.D., Chief Business Officer
Oct. 4 | 2:30pm | La Jolla Ballroom 1
Houston, TX
(NASDAQ: BLCM)
Bellicum is a clinical-stage biopharmaceutical company focused on discovering and developing cellular immunotherapies for cancers and orphan inherited blood disorders. Bellicum is using its proprietary Chemical Induction of Dimerization (CID) technology platform to engineer and control components of the immune system. Bellicum is developing next-generation product candidates in some of the most important areas of cellular immunotherapy, including hematopoietic stem cell transplantation (HSCT) and CAR-T and TCR cell therapies. www.bellicum.com

Benitec_150x65

BENITEC BIOPHARMA
David Suhy, Ph.D., Chief Scientific Officer
Oct. 5 | 3:45pm | La Jolla Ballroom 2
Sydney, Australia
(NASDAQ: BNTC; ASX: BLT)
Benitec Biopharma is developing a proprietary therapeutic technology platform that combines gene silencing and gene therapy with a goal of providing sustained, long- lasting silencing of disease-causing genes from a single administration. The company’s technology platform, called DNA-directed RNA interference, (ddRNAi) is designed to utilize the specificity and gene silencing effect of RNAi while overcoming many of the limitations of siRNA. Benitec’s ddRNAi approach combines RNA interference with gene therapy vectors to deliver a DNA compound to the target diseased tissue in order to silence the disease-associated genes. www.benitec.com

BioTime_140x65

BIOTIME
James Knight, SVP, Head of Corporate Development
Oct. 5 | 4:45pm | La Jolla Ballroom 1
Alameda, CA
(NASDAQ: BTX)
BioTime is a clinical-stage biotechnology company developing and commercializing products addressing degenerative diseases. BioTime has two main technology platforms: a premier collection of pluripotent cell assets and the HyStem hydrogel platform. BioTime currently focuses its development efforts on ophthalmology, aesthetics and the delivery of cells and biomolecules with HyStem. BioTime also has significant equity holdings in two publicly traded companies, Asterias Biotherapeutics and OncoCyte Corporation, which BioTime founded. www.biotimeinc.com

Bluebird-Bio_160x65

BLUEBIRD BIO
Jeffrey Walsh, Chief Financial and Strategy Officer
Oct. 4 | 3:00pm | La Jolla Ballroom 1
Cambridge, MA
(NASDAQ: BLUE)
With its lentiviral-based gene therapies, T-cell immunotherapy expertise and gene editing capabilities, bluebird bio has built an integrated product platform with broad potential application to severe genetic diseases and cancer. bluebird bio’s gene therapy clinical programs include its Lenti-D™ product candidate for the treatment of cerebral adrenoleukodystrophy and its LentiGlobin™ BB305 product candidate for the treatment of transfusion-dependent β-thalassemia and severe sickle cell disease. bluebird bio’s oncology pipeline is built upon the company’s leadership in lentiviral gene delivery and T-cell engineering. bluebird bio’s lead oncology program, bb2121, is an anti-BCMA CAR-T program partnered with Celgene. bluebird bio also has discovery research programs utilizing megaTALs/homing endonuclease gene editing technologies with the potential for use across the company’s pipeline. www.bluebirdbio.com

BlueRock_160x65

BLUEROCK THERAPEUTICS
Emile Nuwaysir, Ph.D., CEO
Oct. 4 | 4:15pm | La Jolla Ballroom 2
Cambridge, MA
(Private)
Driven by a vision to liberate patients from the burden of degenerative disease, BlueRock Therapeutics is ushering in a new era of cell-based medicine that repairs the body when it cannot repair itself. Founded in 2016 through one of the largest Series A financings in biotech history, BlueRock and its team of preeminent scientists are pioneering cell therapies that replace dead, damaged or dysfunctional cells to restore critical natural functions in the body. Using an approach that can be applied to multiple diseases with great unmet need, BlueRock is initially targeting severe brain and heart conditions, with the goal of altering the course of disease and drastically improving quality of life. BlueRock’s culture is defined by scientific innovation, highest ethical standards and an urgency to bring transformative treatments to all who would benefit. www.bluerocktx.com

B-Mogen_130x65

B-MOGEN BIOTECHNOLOGIES
Branden Moriarity, Ph.D., Chief Scientific Officer
Oct. 5 | 3:15pm | La Jolla Ballroom 2
Minneapolis, MN
(Private)
B-MoGen Biotechnologies is a genome editing company that was born out of a necessity for better biological research tools. The company has developed innovative methods that employ cutting edge and time-tested genome editing technologies to study human disease and genetics. B-Mogen offers unique gene editing solutions for antibody validation, human disease research, gene transfer and mitochondrial DNA editing. www.bmogen.com

Bone_170x65

BONE THERAPEUTICS
Miguel Forte, M.D., Ph.D., Chief Medical Officer
Oct. 4 | 2:15pm | La Jolla Ballroom 2
Gosselies, Belgium
(EURONEXT BRUSSELS; EURONEXT PARIS: BOTHE)
Bone Therapeutics is a leading cell therapy company addressing high unmet needs in orthopedics and bone diseases. The company has a broad, diversified portfolio of bone cell therapy products in clinical development across a number of disease areas targeting markets with large unmet medical needs and limited innovation. Bone Therapeutics’ technology is based on a unique, proprietary approach to bone regeneration which turns undifferentiated stem cells into “osteoblastic” or bone-forming cells. These cells can be administered via a minimally invasive procedure, avoiding the need for invasive surgery. The company’s primary clinical focus is ALLOB®, an allogeneic off-the-shelf cell therapy product derived from stem cells of healthy donors, which is in Phase II studies for the treatment of delayed-union fractures and spinal fusion. The company also has an autologous bone cell therapy product, PREOB®, obtained from patients’ own bone marrow and currently in Phase III development for osteonecrosis and non-union fractures. www.bonetherapeutics.com

Caladrius_130x65

CALADRIUS BIOSCIENCES
David Mazzo, Ph.D., President and CEO
Oct. 5 | 9:15am | La Jolla Ballroom 2
Basking Ridge, NJ
(NASDAQ: CLBS)
Caladrius Biosciences is a clinical-stage development company with cell therapy products in development based on multiple technology platforms and targeting autoimmune and select cardiology indications. The company is investigating its lead product candidate, CLBS03, an ex-vivo expanded polyclonal T regulatory cell therapy for the treatment of recent-onset Type 1 diabetes in a currently enrolling Phase II trial. www.caladrius.com

Capricor_145x65

CAPRICOR THERAPEUTICS
AJ Bergmann, VP, Finance
Oct. 4 | 11:15am | La Jolla Ballroom 2
Los Angeles, CA
(NASDAQ: CAPR)
Capricor Therapeutics is a clinical-stage biotechnology company developing first-in-class biological therapies. Capricor’s lead candidate, CAP-1002, is a cell-based candidate currently in clinical development for the treatment of Duchenne muscular dystrophy. Capricor is also exploring the potential of CAP-2003, a cell-free, exosome-based candidate, to treat a variety of disorders. www.capricor.com

CARsgen_150x65

CARSGEN THERAPEUTICS
Cindy Ru, Ph.D., Chief Scientific Officer
Oct. 4 | 5:00pm | La Jolla Ballroom 1
San Diego, CA
(Private)
Deeply rooted in China and growing to be a global leader, CARsgen is a clinical stage CAR-T therapy biotech startup, committed to fulfilling unmet medical needs. The company is specifically focused on orphan indications in the western world that also commonly occur in China and the Asia Pacific. www.carsgen.com

Cell-Medica_155x65

CELL MEDICA
Stefanos Theoharis, Ph.D., SVP, Corporate Development and Partnering
Oct. 5 | 1:30pm | La Jolla Ballroom 1
London, UK
(Private)
Cell Medica is committed to improving patients’ lives through the significant therapeutic potential of cellular immunotherapy. The company’s approach is to apply innovative technologies with the aim of improving the treatment of cancer and immune reconstitution following hematopoietic stem cell transplant. Cell Medica has developed a business platform that has positioned the company as a leader in the manufacture and commercialization of cellular products. Cell Medica collaborates with leading academic institutions to turn early-stage innovations into medicinal products. The company’s highly dedicated team embrace the challenges that the field of cellular therapies presents. With patient benefits at the forefront of Cell Medica’s activities, the company overcomes considerable challenges through collaboration and the will to succeed. www.cellmedica.com

CellSeed_140x65

CELLSEED
Setsuko Hashimoto, Ph.D., President and CEO
Oct. 5 | 1:45pm | La Jolla Ballroom 2
Tokyo, Japan
(JASDAQ Growth: 7776)
CellSeed is proud of the company’s flagship product, the cell sheet, born of an innovative concept which originated in Japan from a platform technology in the field of regenerative medicine since its establishment. In 2015, CellSeed established an overseas subsidiary called CellSeed Sweden AB in Stockholm, Sweden. The company conducts research and development, marketing and sales of regenerative medicine products in Europe. It is already in preliminary consultations with the European Medical Agency (EMA) to acquire marketing authorization across Europe for the epithelial cell sheet for esophageal regeneration for its application on healing post-operative lesions resulting from superficial esophageal cancer. In 2016, CellSeed started clinical trials in Japan on the epithelial cell sheet for esophageal regeneration. Also in 2016, the company moved its headquarters to the Telecom Center Building in the Odaiba Waterfront Area, constructing a cell processing center (CPC) in the same building and creating a good foothold for the commercialization of the cell sheet business. www.cellseed.com

Cellular-Dynamics-International_135x65

CELLULAR DYNAMICS INTERNATIONAL
Kazuyoshi Hirao, Chairman and CEO
Oct. 4 | 4:30pm | La Jolla Ballroom 2
Madison, WI
(NASDAQ: FUJIY)
Cellular Dynamics International (CDI), a FUJIFILM company, is the leading developer and manufacturer of iPSC-derived human cells used in drug discovery, toxicity testing, stem cell banking and cell therapy development. Leveraging technology that can be used to create induced pluripotent stem cells (iPSCs) and differentiated tissue-specific cells from any individual, CDI is committed to advancing life science research and transforming the therapeutic development process to fundamentally improve human health. In addition to the commercially available iCell® and MyCell® products, the company has four internal cell therapy programs in preclinical development (ocular, cardiac, Parkinson’s disease and CAR-T/NK). With expanding cGMP resources, CDI employs industry-leading process development and manufacturing capabilities to also offer contract development and manufacturing services for partner cell therapy programs. www.cellulardynamics.com

Cesca_140x65

CESCA THERAPEUTICS
Phil Coelho, Chief Technology Officer
Oct. 4 | 11:30am | La Jolla Ballroom 2
Rancho Cordova, CA
(NASDAQ: KOOL)
Cesca is a leading regenerative medicine company that develops, commercializes and markets a range of automated technologies for cell-based therapeutics. Its device division provides a full suite of solutions for automated clinical biobanking, point-of-care applications and automation for immuno-oncology. Cesca is also leveraging its proprietary AutoXpress® technology platform to develop autologous stem cell-based therapies that address significant unmet needs in the vascular, cardiology and orthopedic markets. Cesca is an affiliate of the BoyaLife Group, a China-based industry research alliance encompassing top research institutions for stem cell and regenerative medicine. www.cescatherapeutics.com

CRISPR_65x65

CRISPR THERAPEUTICS
Bill Lundberg, M.D., Chief Scientific Officer
Oct. 4 | 11:45am | La Jolla Ballroom 1
Boston, MA
(NASDAQ: CRSP)
CRISPR Therapeutics is a leading gene editing company focused on the development of transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 gene editing platform. CRISPR/Cas9 is a revolutionary technology that allows for precise, directed changes to genomic DNA. The company’s multi-disciplinary team of world-class researchers and drug developers is working to translate this technology into breakthrough human therapeutics in a number of serious diseases. In addition to CRISPR’s fully-owned programs, the company has established strategic collaborations with Bayer AG and Vertex Pharmaceuticals to develop CRISPR-based therapeutics in other diseases with high unmet need. The company has licensed the foundational CRISPR/Cas9 patent estate for human therapeutic use from CRISPR Therapeutics’ scientific founder, Emmanuelle Charpentier, who co-invented the application of CRISPR/Cas9 for gene editing. www.crisprtx.com

Cynata_140x65

CYNATA THERAPEUTICS
Ross Macdonald, Ph.D., CEO
Oct. 4 | 4:00pm | La Jolla Ballroom 2
Melbourne, Australia
(ASX: CYP)
Cynata Therapeutics is an Australian clinical-stage stem cell and regenerative medicine company that is developing a therapeutic stem cell platform technology, Cymerus™, originating from the University of Wisconsin-Madison, a world leader in stem cell research. The proprietary Cymerus™ technology addresses a critical shortcoming in existing methods of production of mesenchymal stem cells (MSCs) for therapeutic use, which is the ability to achieve economic manufacture at commercial scale. Cymerus™ utilizes induced pluripotent stem cells (iPSCs) to produce therapeutic MSCs and the company is the first in the world to conduct a clinical trial with an allogeneic iPSC-derived product. The Cymerus™ platform provides a source of MSCs that is independent of donor limitations and provides an “off-the-shelf” stem cell platform for therapeutic product use. This creates a new standard in stem cell therapeutics and provides both a unique differentiator and an important competitive position. www.cynata.com

DiscGenics_165x65

DISCGENICS
Flagg Flanagan, Chairman and CEO
Oct. 4 | 2:30pm | La Jolla Ballroom 2
Salt Lake City, UT
(Private)
DiscGenics is a biotechnology company developing and commercializing advanced spinal therapeutics to treat patients with diseases of the intervertebral disc. With back pain being such a significant problem globally and a burden on the healthcare system, DiscGenics’ technology has the potential to increase quality of life for patients and to reduce costs to the healthcare system, while providing a significant revenue opportunity. DiscGenics is the only company to utilize allogeneic therapeutic progenitor cells derived from intervertebral disc to treat the disc. The company’s first product, Injectable Discogenic Cell Therapy (IDCT), is starting clinical testing for the treatment of moderate degenerative disc disease (DDD). www.discgenics.com

Fate_85x65

FATE THERAPEUTICS
Dan Shoemaker, Ph.D., Chief Scientific Officer
Oct. 4 | 2:15pm | La Jolla Ballroom 1
San Diego, CA
(NASDAQ: FATE)
Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders. The company’s hematopoietic cell therapy pipeline is comprised of NK- and T-cell immuno-oncology programs, including off-the-shelf product candidates derived from engineered induced pluripotent cells, and immuno-regulatory programs, including product candidates to prevent life-threatening complications in patients undergoing hematopoietic cell transplantation and to promote immune tolerance in patients with autoimmune disease. Its adoptive cell therapy programs are based on the company’s novel ex-vivo cell programming approach, which it applies to modulate the therapeutic function and direct the fate of immune cells. www.fatetherapeutics.com

Fibrocell_165x65

FIBROCELL
John Maslowski, CEO
Oct. 4 | 3:00pm | La Jolla Ballroom 2
Exton, PA
(NASDAQ: FCSC)
Fibrocell is an autologous cell and gene therapy company translating personalized biologics into medical breakthroughs for diseases affecting the skin and connective tissue. Fibrocell’s most advanced product candidate, FCX-007, is the subject of a Phase I/II clinical trial for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). Fibrocell is in pre-clinical development of FCX-013, its product candidate for the treatment of moderate to severe localized scleroderma. Fibrocell’s gene therapy portfolio is being developed in collaboration with Intrexon Corporation (NYSE: XON), a leader in synthetic biology. www.fibrocell.com

GammaDelta_175x65

GAMMADELTA THERAPEUTICS
Natalie Mount, Ph.D., Chief Scientific Officer
Oct. 5 | 2:00pm | La Jolla Ballroom 2
London, UK
(Private)
Gamma delta T-cells are a unique and conserved population of lymphocytes that contribute to many types of immune responses and immunopathologies. GammaDelta Therapeutics is a new spinout company headquartered in London and based on pioneering research conducted at King’s College and the Francis Crick Institute in the group of Professor Adrian Hayday, enabling for the first time the ability to isolate large numbers of high quality, human tissue-resident gamma delta T-cells. The company is focused on exploiting the unique characteristics of these cells to develop improved therapies for cancer and autoimmune diseases. www.gammadeltatx.com

Healios_150x65

HEALIOS
Hardy TS Kagimoto, M.D., Ph.D, President and CEO
Oct. 4 | 4:45pm | La Jolla Ballroom 2
Tokyo, Japan
(4593: Tokyo)
Healios is a biotechnology venture leading the field in developing iPS cell-based products for regenerative medicine. The company was founded in 2011 and listed on the stock exchange in 2015. Helios is developing a treatment for age-related macular degeneration (AMD) and organ bud transplantation therapy involving a technology to create functional human organs as an iPSC pipeline. The company is conducting a Phase II/III study in Japan for ischemic stroke as a somatic stem cell pipeline. www.healios.co.jp/en/

Histogenics_160x65

HISTOGENICS
Adam Gridley, President and CEO
Oct. 4 | 3:15pm | La Jolla Ballroom 2
Waltham, MA
(NASDAQ: HSGX)
Histogenics is a leading regenerative medicine company developing and commercializing novel tissue therapies that may offer more rapid and durable recoveries for patients with pain and loss of function due to musculoskeletal conditions. Histogenics’ regenerative medicine platform combines expertise in cell processing, scaffolding, tissue engineering and bio-adhesives to create tissue ex-vivo. Histogenics’ first investigational product candidate, NeoCart® is designed to treat cartilage defects in the knee and is currently in Phase III clinical development. NeoCart® is designed to exhibit characteristics of articular, hyaline cartilage prior to and upon implantation into the knee and therefore does not rely on the body to make new cartilage. As a result, NeoCart® is the only product in development or on the market with a one-year primary superiority endpoint as compared to the standard of care. www.histogenics.com

Homology_150x65

HOMOLOGY MEDICINES
Arthur Tzianabos, Ph. D., CEO
Oct. 5 | 11:15am | La Jolla Ballroom 2
Bedford, MA
(Private)
Homology Medicines is a genetic medicines company translating proprietary gene editing and gene therapy technologies into novel treatments for patients with rare diseases. The combination of a new multidimensional technology platform and a management team that has successfully developed and commercialized rare disease therapies uniquely positions the company to move beyond the current limitations of gene therapy and gene editing approaches to improve patient care. Homology has built foundational intellectual property on gene editing and gene therapy using vectors derived from naturally occurring human adeno-associated viruses (AAVs). The company’s technology is precise, on-target and highly efficient for in vivo editing of genetic mutations. The company’s unique team and technology create a significant opportunity for Homology to rapidly advance a diverse pipeline of new medicines that address and potentially cure the underlying cause of genetic diseases. www.homologymedicines.com

Intellia_110x65

INTELLIA THERAPEUTICS
Jenn King, Ph.D., SVP, Business Development
Oct. 5 | 1:45pm | La Jolla Ballroom 1
Cambridge, MA
(NASDAQ: NTLA)
Intellia Therapeutics is a leading genome editing company focused on the development of proprietary, potentially curative therapeutics using the CRISPR/Cas9 system. Intellia believes CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course. Intellia’s combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts it in a unique position to unlock broad therapeutic applications of CRISPR/Cas9 technology and create a new class of therapeutic products. www.intelliatx.com

jCyte_80x65

JCYTE
Henry Klassen, M.D., Ph.D., President
Oct. 4 | 5:30pm | La Jolla Ballroom 2
Newport Beach, CA
(Private)
jCyte is a clinical-stage company focused on the application of patented progenitor cell-based technology in retinal diseases. The company’s allogeneic jCell product can be delivered by simple intravitreal injection without the need for surgery or immune suppression. A Phase I/IIa trial in retinitis pigmentosa (RP) has enrolled 28 patients and completion is expected in August, 2017. Initial data indicate that jCell is well tolerated without immune suppression. Preliminary indications of activity have been sufficient to apply to the FDA for regenerative medicine advanced therapy (RMAT) designation, which was granted in April 2017. An extension trial is underway that includes dosing of the fellow eye and a follow-on trial is in place to demonstrate clinical proof-of-concept. jCyte is in the process of defining an efficient path to approval in partnership with the FDA and additional retinal disease indications for jCell are currently under investigation. www.jcyte.com

Juventas_115x65

JUVENTAS THERAPEUTICS
Rahul Aras, Ph.D., President and CEO
Oct. 4 | 11:00am | La Jolla Ballroom 2
Cleveland, OH
(Private)
Juventas Therapeutics is a private, clinical-stage biotechnology company developing novel non-viral gene therapies that activate natural processes to repair the body. The company’s product candidate, JVS-100, is a non-viral gene therapy that expresses stromal cell-derived factor-1, or SDF-1, a naturally occurring signaling protein that has been shown to recruit the body’s own stem cells and promote tissue repair in a broad range of disease states. Juventas’ therapeutic approach is based on research originating at the Cleveland Clinic. The company is currently enrolling a Phase IIb study in patients with advanced peripheral artery disease. www.juventasinc.com

LogicBio_130x65

LOGICBIO THERAPEUTICS
Fred Chereau, CEO
Oct. 4 | 11:00am | La Jolla Ballroom 2
Cambridge, MA
(Private)
Our mission is to develop lifelong cures for serious, early-onset rare diseases by combining the best of gene therapy and gene editing in a one-time treatment. With GeneRide, the therapeutic gene is site-specifically integrated into the genome allowing durable gene expression in dividing cells and mature tissues. Furthermore, therapeutic gene expression is regulated by the targeted locus so production can be limited to specific cell types. GeneRide is a promoterless and nuclease-free approach to gene insertion which may improve the safety profile of AAV-based gene therapies. www.logicbio.com

Lysogene_140x65

LYSOGENE
Sean O’Bryan, VP, Regulatory Affairs and Quality Control
Oct. 4 | 11:45am | La Jolla Ballroom 2
Neuilly-sur-Seine, France
(EURONEXT PARIS: LYS)
Lysogene is a clinical-stage biotechnology company pioneering the basic research and clinical development of AAV gene therapy for CNS disorders with a high unmet medical need. Since 2009, Lysogene has established a solid platform and network with lead products in mucopolysaccharidosis type IIIA and GM1 gangliosidosis, to become a global leader in orphan CNS diseases. Neurological disorders such as MPS IIIA and GM1 require therapy to be broadly distributed throughout the brain. Since the blood brain barrier prevents efficient uptake of gene therapy vectors from the systemic circulation, Lysogene’s technology delivers vectors directly to the CNS. Lysogene has obtained orphan drug designation by the EMA and FDA and rare pediatric designation by the FDA for its MPS IIIA and GM1 programs. www.lysogene.com

MaxCyte_170x65

MAXCYTE
Madhusudan Peshwa, Ph.D., Chief Scientific Officer
Oct. 4 | 3:30pm | La Jolla Ballroom 2
Gaithersburg, MD
(Private)
MaxCyte is a U.S.-based global company dedicated to accelerating the discovery, development, manufacturing and commercialization of next-generation, cell-based medicines. The company provides its patented, high-performance cell engineering platform to biopharmaceutical partners engaged in drug discovery and development, biomanufacturing and cell therapy, including gene editing and immuno-oncology. With its robust delivery platform, MaxCyte’s team of scientific experts helps its partners to unlock their product potential and solve development and commercialization challenges. This platform allows for the engineering of nearly all cell types, including human primary cells, with any molecule, at any scale. It also provides unparalleled consistency and minimal cell disturbance, thereby facilitating rapid, large-scale, clinical and commercial grade, high-performance cell engineering in a non-viral system and with low-toxicity concerns. The company’s cell-engineering platform is CE-marked and FDA-accredited, providing MaxCyte’s customers and partners with an established regulatory path to commercialize cell-based medicines. www.maxcyte.com

Medeor_130x65

MEDEOR THERAPEUTICS
Steven Deitcher, M.D., Co-founder, President and CEO
Oct. 4 | 1:30pm | La Jolla Ballroom 2
San Mateo, CA
(Private)
Medeor Therapeutics is a private, late clinical stage, biotechnology company dedicated to the discovery, development, and commercialization of transformative and personalized cellular immunotherapies. Our growing team of professionals possesses remarkably deep expertise in cellular immunotherapy and transplantation product development. Our pipeline addresses critical medical needs of the approximately 120,000 annual organ transplant recipients worldwide, including greater than 30,000 annual organ transplant recipients in the U.S. alone. Medeor’s product candidates are intended to minimize or eliminate chronic anti-rejection, immunosuppressive drug regimens used by new recipients of living donor transplants as well as those who have received a living donor transplant in the past (i.e., 2nd Chance Therapy). We hold exclusive worldwide licenses to a series of breakthrough technologies, all discovered at Stanford University. www.medeortherapeutics.com

MEDIPOST_86x65

MEDIPOST AMERICA
Antonio Lee, Ph.D., CEO
Oct. 4 | 2:45pm | La Jolla Ballroom 2
Rockville, MD
(Private)
MEDIPOST develops allogeneic stem cell products using multi-potent human Umbilical Cord Blood-derived Mesenchymal Stem Cells (hUCB-MSCs). MEDIPOST also operates the largest Cord Blood Bank – CellTree™, in Korea with over 220,000 units of cord blood stored, covering over 45% of private cord blood banking market-share in Korea. MEDIPOST America is a wholly-owned U.S. subsidiary. MEDIPOST has introduced the world’s first allogeneic off-the-shelf hUCB-MSC product – CARTISTEM®, into the Korean market and approved by the Ministry of Food and Drug Safety for patients with knee osteoarthritis (OA) across all ages. CARTISTEM® completed Phase I/II and III clinical trials in Korea leading to market-approval in 2012. CARTISTEM® also completed a Phase I/II clinical trial in the U.S. MEDIPOST’s other pipeline of allogeneic hUCB-MSC products target broncho-pulmonary dysplasia (BPD) in premature infants (Phase I and II complete in Korea; Phase I/II complete in the U.S.) and Alzheimer’s disease (Phase I complete in Korea). www.medipostamerica.com

Mesoblast_175x65

MESOBLAST
Michael Schuster, Head of Pharma Partnering
Oct. 4 | 5:30pm | La Jolla Ballroom 1
Melbourne, Australia
(NASDAQ: MESO; ASX: MSB)
Mesoblast Limited is building a leading franchise of cellular medicines to treat serious and life-threatening illnesses. The company has leveraged its proprietary disruptive technology platform, based on allogeneic off-the-shelf mesenchymal lineage adult stem cells, to target advanced stages of diseases with high, unmet medical needs including cardiovascular diseases, immune-mediated and inflammatory conditions, spine orthopedic disorders and oncology/hematology diseases. Each of these products has its own distinct technical characteristics, target indications, separate commercialization potential and unique partnering opportunities. In 2016, Mesoblast’s licensee in Japan, JCR Pharmaceuticals, received the first full approval for an allogeneic cellular medicine in Japan. www.mesoblast.com

MolMed_130x65

MOLMED
Luca Alberici, Ph.D., Business Development Director
Oct. 4 | 3:15pm | La Jolla Ballroom 1
Milan, Italy
(MLM.MI)
MolMed S.p.A. is a medical biotechnology company focused on research, development and clinical validation of novel cell and gene therapy products. MolMed’s pipeline main products are Zalmoxis® (TK), a cell-based therapy in Phase III in high-risk acute leukemia and granted a Conditional Marketing Authorization by the European Commission, and CAR-CD44v6, an immune gene therapy project potentially effective for many hematological malignancies and several epithelial tumors, currently in preclinical development. MolMed also offers top-level expertise in cell and gene therapy to third parties to develop and validate projects from preclinical to Phase III trials, including scale-up and cGMP manufacturing of retroviral and lentiviral vectors and patient-specific genetically engineered CD34+ or T-cells. MolMed is headquartered in Milan and has two GMP manufacturing facilities in the Milan area. MolMed has been listed on the Milan Stock Exchange managed by Borsa Italiana since 2008. www.molmed.com

NYSCF_100x65

THE NEW YORK STEM CELL FOUNDATION
Stephen Chang, Ph.D., Scientific Advisor
Oct. 5 | 11:45am | La Jolla Ballroom 2
New York, NY
(Private)
The New York Stem Cell Foundation (NYSCF) is an independent, nonprofit organization founded in 2005 to accelerate cures and better treatments for patients through stem cell research. NYSCF employs over 45 researchers at the NYSCF Research Institute, located in New York City, and is an acknowledged world leader in stem cell research and in developing pioneering stem cell technologies, including the NYSCF Global Stem Cell Array. Additionally, NYSCF supports over 75 researchers at other leading institutions worldwide through its Innovator Programs, including the NYSCF – Druckenmiller Fellowships and the NYSCF – Robertson Investigator Awards. NYSCF focuses on translational research in a model designed to overcome the barriers that slow discovery and replaces silos with collaboration. www.nyscf.org

Orbsen_210x65

ORBSEN THERAPEUTICS
Larry Couture, Ph.D., CEO
Oct. 5 | 10:00am | La Jolla Ballroom 2
Galway, Ireland
(Private)
Orbsen Therapeutics is a stromal cell immunotherapy company based in Galway, Ireland. Using proprietary GMP-compliant and scalable positive-selection purification strategies, the company is developing groundbreaking, second-generation stromal cell treatments to address some of today’s most challenging diseases. Stromal cell immunotherapy holds great promise as an alternative to drugs and surgical procedures for treating a wide range of medical conditions including acute inflammatory disease, arterial disease of the limbs, diabetes complications, arthritis and other inflammatory conditions. This treatment potential is linked to stromal cells’ natural capacity to modulate the immune system, limit inflammation and promote healing, repair and regeneration of damaged tissues. The cells can be selected from single or pooled donors and expanded and frozen to generate significant numbers of well characterized, “off-the-shelf” allogeneic products. Clinical trials in patients suffering from diabetic foot ulcers, autoimmune liver disease, diabetic kidney disease and acute respiratory distress syndrome are beginning. www.orbsentherapeutics.com

Orchard_115x65

ORCHARD THERAPEUTICS
Mark Rothera, CEO
Oct. 5 | 11:30am | La Jolla Ballroom 2
London, UK
(Private)
Orchard Therapeutics is a biotechnology company incorporated in September 2015 and dedicated to bringing transformative gene therapies to patients with serious and life-threatening orphan diseases. The company’s programs will use the potential of ex-vivo autologous hematopoietic stem cell gene therapy to restore normal gene function in severe and life-threatening inherited disorders. Orchard works in partnership with the world’s leading research centers, and the company’s mission is to translate results from preclinical and early clinical research into commercially approved gene therapies for patients around the world. Orchard’s current focus is primary immune-deficiencies and lysosomal storage disorders. The company’s lead program, OTL-101 for ADA-SCID, is in late clinical-stage with very strong results accrued to date. Orchard Therapeutics is a 2016 “Fierce 15” Fierce Biotech company. www.orchard-tx.com

Oxford-BioMedica_170x65

OXFORD BIOMEDICA
Peter Nolan, Chief Business Officer
Oct. 5 | 4:15pm | La Jolla Ballroom 1
Oxford, UK
(LON: OXB)
Oxford BioMedica has 20 years of experience in the field of gene and cell therapy and was the first organization to treat humans in vivo lentiviral based vectors. Today, Oxford BioMedica has built the company’s LentiVector® platform of exclusive cutting-edge technologies and capabilities with which the company designs, develops and produces gene and cell-based medicines for Oxford BioMedica itself and for partners. The company already has product-related partnerships with Novartis, Immune Design, Orchard Therapeutics, licensed products and technology rights to Sanofi and GSK and a research and development collaboration with Green Cross LabCell. In addition, Oxford BioMedica has its own proprietary pipeline of gene and cell therapy products addressing neurodegenerative and ocular diseases and a range of cancers, for which there are either no treatments or where therapy remains inadequate. www.oxfordbiomedica.co.uk

Precision-Biosciences_145x65

PRECISION BIOSCIENCES
Michael Dombeck, VP, Business Development
Oct. 5 | 1:15pm | La Jolla Ballroom 1
Durham, NC
(Private)
Precision BioSciences is a privately-held biotechnology company dedicated to improving lives through its next generation gene editing technology, ARCUS. Precision’s approach is based on a proprietary synthetic enzyme, the ARC endonuclease, which features coordinated target site binding and DNA cutting to ensure precise editing outcomes. Uniquely small and monomeric, ARC nucleases are readily delivered to target sites throughout the genome and across cell types and tissues. The value of the ARCUS editing platform is enhanced by Precision’s extensive patent portfolio, established freedom to operate and deep collaborative relationships, positioning Precision to create products that solve significant problems in oncology, genetic disease, agriculture and beyond. The team includes pioneers in genome engineering and a staff of experienced, committed Precisioneers. Working together in the vibrant innovation center of Durham, NC, Precision is excited to lead the next wave of medical and scientific possibilities through continuous gene editing innovation. www.precisionbiosciences.com

Promethera_155x65

PROMETHERA BIOSCIENCES
Mark Johnston, COO and Head of U.S. Operations
Oct. 4 | 1:45pm | La Jolla Ballroom 2
Mont-Saint-Guibert, Belgium
(Private)
Promethera Biosciences is a global innovator in liver cell-based medicines whose mission is to help patients overcome acute and chronic liver diseases. The company’s lead clinical program, derived from Promethera’s patented cell technology platform HepaStem, is designed to benefit from its immune-modulatory and anti-fibrotic properties. Promethera is a team of international experts operating out of research and development and GMP facilities in Mont-Saint-Guibert, Belgium and Durham, NC. www.promethera.com

REGENXBIO_170x65

REGENXBIO
Faraz Ali, Chief Business Officer
Oct. 5 | 2:00pm | La Jolla Ballroom 1
Rockville, MD
(NASDAQ: RGNX)
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO’s NAV® Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Licensees are applying the NAV Technology Platform in the development of a broad pipeline of product candidates in multiple therapeutic areas. REGENXBIO is currently developing gene therapy product candidates in the retinal, metabolic and neurodegenerative therapeutic areas. To date, there are over 20 partnered programs – including seven clinical-stage programs – being developed by nine NAV Technology Licensees, including Adverum Biotechnologies, Audentes Therapeutics, AveXis, Biogen, Dimension Therapeutics, Esteve, Lysogene, Shire and Voyager Therapeutics. www.regenxbio.com

ReNeuron_135x65

RENEURON
Michael Hunt, Chief Financial Officer
Oct. 5 | 5:15pm | La Jolla Ballroom 1
Bridgend, Wales
(LSE: RENE)
ReNeuron is a leading, clinical-stage cell therapy development company. ReNeuron has therapeutic candidates in clinical development for motor disability as a result of stroke, for critical limb ischaemia and for the blindness-causing disease, retinitis pigmentosa. ReNeuron is also advancing its proprietary exosome technology platform as a potential new nanomedicine targeting cancer and as a potential delivery system for gene therapy treatments. www.reneuron.com

Renova_115x65

RENOVA THERAPEUTICS
Jack Reich, Ph.D., Co-Founder and CEO
Oct. 4 | 10:45am | La Jolla Ballroom 2
San Diego, CA
(Private)
Founded in 2009 by pioneers in biopharmaceuticals, Renova Therapeutics is working to create a portfolio of definitive gene and peptide therapies to restore health to people suffering from cardiovascular and metabolic diseases. The first indications the company is pursuing are gene therapies for congestive heart failure (CHF) and Type 2 diabetes, two of the most common and devastating chronic diseases in the world. Along with single-dose gene therapies, Renova Therapeutics is also developing a peptide infusion treatment for the periods in which CHF patients are hospitalized for acute decompensated heart failure. The company’s mission is to create transformational gene and peptide therapies that treat the most prevalent diseases to restore health and renew life. Renova’s vision is a future in which gene and peptide therapies are able to permanently treat chronic diseases. www.renovatherapeutics.com

RoosterBio_175x65

ROOSTERBIO
Jon Rowley, Ph.D., Founder and Chief Technology Officer
Oct. 5 | 5:15pm | La Jolla Ballroom 2
Frederick, MD
(Private)
Stem cells have created a paradigm shift in the world of medicine, moving from symptomatic treatments to one capable of curing diseases. RoosterBio is an innovative regenerative medicine (RM) technology company dedicated to driving the acceleration of stem cell-based therapeutics and supporting the development of a sustainable regenerative medicine industry. RoosterBio is a privately-held Maryland-based company focused on manufacturing and supplying stem cell systems in product configurations that enable 3D bioprinting, manufacturing scale-up and clinical translation. The company’s products are high volume, efficient and well-characterized adult hMSCs paired with highly engineered media systems. RoosterBio is taking a unique business approach towards regenerative medicine that will revolutionize how therapies and devices that require living cells are commercialized. The company’s products are designed to address a significant bottleneck in RM product development and removes several years and millions of dollars from the product development and clinical testing cycles. www.roosterbio.com

Sangamo_140x65

SANGAMO THERAPEUTICS
Sandy Macrae, Ph.D., CEO
Oct. 5 | 2:15pm | La Jolla Ballroom 1
Richmond, CA
(NASDAQ: SGMO)
Sangamo Therapeutics is focused on translating ground-breaking science into genomic therapies that transform patients’ lives using the company’s industry leading platform technologies in genome editing, gene therapy, gene regulation and cell therapy. The company is conducting Phase I/II clinical trials in Hemophilia A and Hemophilia B, and lysosomal storage disorders MPS I and MPS II. Sangamo has an exclusive, global collaboration and license agreement with Pfizer for gene therapy programs for Hemophilia A, with Bioverativ for hemoglobinopathies, including beta thalassemia and sickle cell disease, and with Shire International GmbH to develop therapeutics for Huntington’s disease. In addition, it has established strategic partnerships with companies in non-therapeutic applications of its technology including Sigma-Aldrich Corporation and Dow AgroSciences. www.sangamo.com

SCM_120x65

SCM LIFESCIENCE
Sun Song, Ph.D., CEO
Oct. 5 | 5:00pm | La Jolla Ballroom 2
Incheon, Korea
(Private)
SCM Lifescience was established with the vision of developing treatment options for difficult to treat diseases, to provide a new source of hope for patients and to contribute to the welfare of humanity as a whole using proprietary stem cell isolation technology and stem cell production technology. Through over 10 years of research and development, SCM Lifescience has obtained intellectual property protection for its high-purity stem cell isolation technology – registered patent in Korea in 2008, U.S. in 2010, Japan in 2012, China in 2013 and the EU in 2014; high-purity stem cell manufacturing technology – U.S. patent in 2014; and treatment of graft-versus-host disease – patents from Japan in 2014, U.S. in 2015 and the EU in 2016. Based on these technical breakthroughs, high-purity stem cell therapy shall be provided to treat various immune diseases such as graft-versus-host disease, acute pancreatitis, atopic dermatitis and Type I diabetes. SCM’s research will also focus on the field of regenerative medicine for technological breakthroughs including the regeneration of skin, cartilage, bone, hair growth and other tissues and organs. www.scmlifescience.com

Semma_110x65

SEMMA THERAPEUTICS
Felicia Pagliuca, Ph.D., Co-Founder and VP, Cell Biology Research and Development
Oct. 5 | 9:30am | La Jolla Ballroom 2
Cambridge, MA
(Private)
Semma Therapeutics was founded to develop transformative therapies for Type 1 diabetes patients. Work in the laboratory of Professor Douglas Melton led to the discovery of a method to generate billions of functional, insulin-producing beta cells in vitro. This breakthrough technology has been exclusively licensed to Semma Therapeutics for the development of a cell-based therapy for diabetes. Ongoing research at Semma Therapeutics is focused on combining these proprietary cells with its state-of-the-art device to provide a true replacement for the missing beta cells in a diabetic patient without the need for immunosuppression. Semma Therapeutics is working to bring new therapeutic options to the clinic and improve the lives of patients with diabetes. The company is headquartered in Cambridge, MA, and was founded in 2015 with financing led by MPM Capital, F-Prime Capital and strategic investors Novartis and Medtronic. www.semma-tx.com

Sentien_62x65

SENTIEN BIOTECHNOLOGIES
Brian Miller, CEO
Oct. 4 | 1:15pm | La Jolla Ballroom 2
Lexington, MA
(Private)
Sentien Biotechnologies is a privately-owned, clinical-stage company developing novel approaches to cell therapy. Sentien’s technology harnesses the power of cell therapy with innovative drug delivery systems to treat a wide range of acute inflammatory diseases. Sentien’s lead product is designed to allow for controlled, sustained delivery of mesenchymal stem cell (MSC) secreted factors. This approach immobilizes the MSCs in an extracorporeal device, allowing for doses of therapeutic factors that are unattainable by direct injection. www.sentienbiotech.com

Sigilon_110x65

SIGILON
Devyn Smith, Ph.D., Chief Strategy Officer and Head of Operations
Oct. 5 | 1:15pm | La Jolla Ballroom 2
Cambridge, MA
(Private)
Sigilon is developing long term treatments for chronic diseases using new biomaterials that can shield implanted cells from immune attack. Treatments based on the Sigilon technology platform include cell implants that act as responsive “dynamic therapeutics,” providing more natural control for diseases currently treated with intermittent injection or infusion. These diseases include genetic disorders, such as hemophilia and enzyme replacement therapies that require protein replacement as well as disorders that require dynamic protein delivery such as Type 1 diabetes. More natural control would restore health and free patients from the need for therapies that are disruptive to quality of life. Sigilon technology originated from research conducted at the Massachusetts Institute of Technology and Harvard Medical School. Based in Cambridge, MA, Sigilon was founded with major support from Flagship Pioneering. Sigilon is also exploring partnership opportunities to maximize the impact of its groundbreaking technology. www.sigilon.com

StemBioSys_170x65

STEMBIOSYS
Robert Hutchens, President and CEO
Oct. 5 | 4:15pm | La Jolla Ballroom 2
San Antonio, TX
(Private)
StemBioSys is a privately-held biomedical company focused on enhancing the growth and delivery of adult stem cells for research, therapeutic or drug discovery applications. Its patented and proprietary technology platforms – licensed from the UT Systems – overcome key obstacles to creating clinically useful stem cell therapies. The centerpiece of these enabling technologies is the HPME® (High Performance Micro Environment) platform. This cell derived three-dimensional microenvironment allows a variety of stem cells to replicate more rapidly, maintain a small cell size and express markers indicative of retained stem cell potency beyond that seen with traditional tissue culture substrates. This year, StemBioSys will announce an advanced version of its cell expansion technology and specialized cell lines enabled by this technology. The company’s products are available for the research market in the U.S. through VWR. StemBioSys is actively seeking collaborations with partners looking for cell expansion technologies. www.stembiosys.com

Synpromics_130x65

SYNPROMICS
David Venables, Ph.D., CEO
Oct. 5 | 3:30pm | La Jolla Ballroom 2
Edinburgh, Scotland
(Private)
Synpromics has developed a unique, market-leading capability for the creation of synthetic promoters which can be designed to drive precision controlled gene function under a variety of biological conditions. Built around the company’s PromPT™ (Promoter Precision Technology) proprietary technology engine, the company can rapidly design, develop and validate novel promoter systems. Custom promoter design parameters include: optimal expression level, tissue specificity, inducibility, size reduction, kill-switch control, environmental and pathologic response or condition specific. Such precision control is not possible with currently used viral or endogenous promoters, however there is a demand for novel promoters to provide increased precision over the control of gene function in gene therapy, cell therapy and gene editing applications to improve product safety and efficacy. www.synpromics.com

Terumo_160x65

TERUMO BCT
Antoinette Gawin, EVP, Global Commercial
Oct. 4 | 5:00pm | La Jolla Ballroom 2
Lakewood, CO
(JPY: TSE)
Terumo BCT, a global leader in blood component, therapeutic apheresis and cellular technologies, is the only company with the unique combination of apheresis collections, manual and automated whole blood processing, and pathogen reduction technologies. Terumo BCT believes in the potential of blood to do even more for patients than it does today. This belief inspires the company’s innovation and strengthens collaboration with customers. www.terumobct.com

TxCell_115x65

TXCELL
Stéphane Boissel, CEO
Oct. 4 | 5:15pm | La Jolla Ballroom 2
Valbonne, France
(EURONEXT PARIS: TXCL)
TxCell is a biotechnology company that develops innovative cell immunotherapies based on regulatory T lymphocytes (Tregs). TxCell is targeting a range of severe inflammatory and autoimmune diseases with high unmet medical needs, including Crohn’s disease, lupus nephritis, bullous pemphigoid and multiple sclerosis, as well as transplant rejection. TxCell is the only clinical-stage cellular therapy company fully dedicated to the science of Tregs. Tregs are a recently discovered T-cell population for which anti-inflammatory properties have been demonstrated. Contrary to conventional approaches based on non-specific polyclonal Tregs, TxCell is exclusively developing antigen-specific Tregs. This antigen specificity may either come from genetic modifications with Chimeric Antigen Receptor (CAR) or from pre-existing Treg T-Cell Receptor (TCR). TxCell is developing two proprietary technology platforms, ENTrIA, which is composed of genetically engineered Tregs, and ASTrIA, which is composed of non-modified naturally antigen-specific Tregs. www.txcell.com

UniversalCells_125x65

UNIVERSAL CELLS
Claudia Mitchell, Ph.D., CEO
Oct. 5 | 4:30pm | La Jolla Ballroom 2
Seattle, WA
(Private)
Universal Cells is developing stem cell therapies that overcome immune rejection and can be used to create truly off-the-shelf stem cell-based therapeutic products. Using the company’s proprietary genome editing approach, Universal Cells engineers stem cells to prevent expression of polymorphic human leukocyte antigen (HLA) molecules, removing the mechanism by which donor tissue rejection occurs and creating Universal Donor Stem Cells that are compatible with every patient. The company has established major strategic partnerships to help advance its Universal Donor Cell Program to the clinic. By combining the safest gene editing approach with a deep understanding of stem cell immunology, the company is positioned to play a central role in the commercialization of pluripotent cell-based therapies. www.universalcells.com

uniQure_140x65

UNIQURE
Jon Garen, Chief Business Officer
Oct. 4 | 3:30pm | La Jolla Ballroom 1
Lexington, MA
(NASDAQ: QURE)
uniQure is delivering on the promise of gene therapy, single treatments with potentially curative results. The company has developed a modular technology platform to rapidly bring new disease-modifying therapies to patients with severe genetic diseases. uniQure is advancing a focused pipeline of innovative gene therapies and have established clinical proof-of-concept in the company’s lead indication, hemophilia B, and preclinical proof-of-concept in Huntington’s disease. uniQure’s pipeline of adeno-associated virus (AAV)-based gene therapies has been developed using an innovative technology platform, supported by industry-leading proprietary commercial-grade manufacturing capabilities. Through recent collaborations and a strategic partnership with Bristol-Myers Squibb to develop gene therapies for cardiovascular diseases, uniQure has taken the next steps toward developing gene therapies targeting chronic and degenerative diseases that affect larger populations. www.uniqure.com

Vericel_160x65

VERICEL
Nick Colangelo, President and CEO
Oct. 4 | 5:45pm | La Jolla Ballroom 1
Cambridge, MA
(NASDAQ: VCEL)
Vericel develops, manufactures and markets autologous cell-based therapies for patients with serious diseases and conditions. The company markets three cell therapy products in the U.S. MACI® is a third generation autologous cellularized scaffold product that is indicated for the repair of single or multiple symptomatic, full-thickness cartilage defects of the adult knee, with or without bone involvement. Carticel® is an autologous chondrocyte implant intended to treat cartilage defects in the knee in patients who have had an inadequate response to a prior arthroscopic or other surgical repair procedure. Epicel® is a permanent skin replacement for the treatment of patients with deep dermal or full thickness burns greater than or equal to 30% of total body surface area. Vericel is also developing one additional cell product. Ixmyelocel-T is a multicellular therapy intended to treat advanced heart failure due to ischemic dilated cardiomyopathy (DCM).
www.vcel.com

VeriGraft_180x65

VERIGRAFT
Petter Bjorquist, Ph.D., CEO
Oct. 5 | 1:30pm | La Jolla Ballroom 2
Gothenburg, Sweden
(Private)
VeriGraft is a Swedish biotechnology company founded as a spin-off from the Karolinska Institute with a unique, breakthrough technology in the field of advanced regenerative medicine. With clinical proof of concept already achieved (results published in the Lancet), VeriGraft is currently preparing for GMP-production and a pivotal clinical trial related to its first product offering, the “P-TEV”, which is a personalized tissue-engineered vein for surgical treatment of chronic venous insufficiency (CVI). The company is expected to sell products and services on a vast market starting with first commercial sales already in 2020. Products will also be developed in other areas of medicine, and VeriGraft’s technology will be able to help millions of patients with serious diseases.

ViaCyte_160x65

VIACYTE
Paul Laikind, Ph.D., President and CEO
Oct. 5 | 9:45am | La Jolla Ballroom 2
San Diego, CA
(Private)
ViaCyte is a privately-held regenerative medicine company developing novel cell replacement therapies as potential long-term diabetes treatments to reduce the risk of hypoglycemia and diabetes-related complications. ViaCyte has two products in clinical development. The PEC-Direct™ product candidate delivers stem cell-derived PEC-01 pancreatic progenitor cells in a non-immunoprotective device and is being developed for Type 1 diabetes patients who have hypoglycemia unawareness, extreme glycemic lability and/or recurrent severe hypoglycemic episodes. The PEC-Encap™ (also known as VC-01) product candidate delivers the same pancreatic progenitor cells in an immunoprotective device, and is being developed for all patients with diabetes, Type 1 and Type 2, who use insulin. The company is funded in part by the California Institute for Regenerative Medicine (CIRM) and JDRF. www.viacyte.com

Vivet_105x65

VIVET THERAPEUTICS
Jean-Philippe Combal, Ph.D., CEO
Oct. 4 | 2:00pm | La Jolla Ballroom 2
Paris, France
(Private)
Vivet Therapeutics is a gene therapy biotech company with headquarters in Paris, France, dedicated to the research, development and future commercialization of gene therapy treatments for inherited liver disorders with high medical need. The company’s development strategy is to target the liver using a novel synthetic adeno-associated virus (AAV-ANC80) to introduce therapeutic genes to hepatocytes, correcting the genetic disorder. Vivet’s keys to success are its exclusive license rights and access to technology granted by its strategic partners, Fundacion Para La Investigacion Medica Aplicada (FIMA) and the Massachusetts Eye and Ear Infirmary (MEE), enabling it to address various inherited disorders such as Wilson’s disease, Progressive Familiar Intrahepatic Cholestasis (PFIC) and Citrullinemia type I in close collaboration with the Center for Applied Medical Research (CIMA) in Pamplona, Spain. www.vivet-therapeutics.com

WindMil_150x65

WINDMIL THERAPEUTICS
Brian Halak, Ph.D., CEO
Oct. 5 | 2:30pm | La Jolla Ballroom 2
Baltimore, MD
(Private)
WindMIL is a clinical-stage biotechnology company developing a new class of cell therapy. Founders Ivan Borrello and Kimberly Noonan from Johns Hopkins University (JHU) have shown that T-cells capable of recognizing and killing tumor cells lie dormant in the bone marrow of cancer patients. These T-cells, known as marrow-infiltrating lymphocytes (MILs), can be extracted from a patient via a routine marrow draw, activated and expanded utilizing a proprietary process, and given back to the patient as an effective and durable therapy. WindMIL has exclusively licensed from JHU the patents and know-how enabling MILs therapy. www.windmiltherapeutics.com