2017 Company Presenters
David Kirn, M.D., Chairman, CEO and Co-Founder
Headquarters: Emeryville, CA
Number of Full-Time Employees: 35
Company Website: www.4dmoleculartherapeutics.com
ABOUT 4D MOLECULAR THERAPEUTICS
4D Molecular Therapeutics (4DMT) is focused on the discovery and development of targeted and proprietary AAV gene therapy vectors and therapeutic products. The company’s robust discovery platform, termed Therapeutic Vector Evolution, empowers 4DMT to create customized gene delivery vehicles to deliver genes to any tissue or organ in the body, by optimal clinical routes of administration and with resistance to pre-existing antibodies. These proprietary and targeted products allow the company to treat both rare genetic diseases and complex large market diseases. 4DMT is creating a diverse and deep product pipeline through partnerships, while progressing internal 4DMT products toward clinical trials in parallel. 4D partners include: Pfizer (PFE), Roche (SIX: ROG; OTCQX: RHHBY), uniQure (QURE), AGTC and Benitec. 4DMT’s lead product is for the treatment of choroideremia, an inherited retinal disease.
Timothy Miller, Ph.D., CEO
Headquarters: Dallas, TX
Number of Full-Time Employees: 22
Company Website: www.abeonatherapeutics.com
February 1, 2017 | Abeona Therapeutics Enrolls First High Dose Subject in Ongoing Phase 1/2 Gene Therapy Clinical Trial in Sanfilippo Syndrome Type A
January 19, 2017 | Abeona Therapeutics Receives Orphan Drug Designation in The European Union for ABO-101 Gene Therapy in Sanfilippo Syndrome Type B
January 3, 2017 | Abeona Therapeutics Receives Orphan Drug Designation in The European Union for ABO-201 Gene Therapy Program in Juvenile Batten Disease
ABOUT ABEONA THERAPEUTICS
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) and ABO-101 (AAV-NAGLU), adeno-associated virus (AAV)-based gene therapies for Sanfilippo syndrome (MPS IIIA and IIIB, respectively). Abeona is also developing EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB), EB-201 for epidermolysis bullosa (EB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JNCL), ABO-202 (AAV-CLN1) gene therapy for treatment of infantile Batten disease (INCL), 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 plasma-based protein therapy pipeline, including SDF Alpha™ (alpha-1 protease inhibitor) for inherited COPD, using its proprietary SD F™ (Salt Diafiltration) ethanol-free process.
Amber Salzman, Ph.D., President and CEO
Headquarters: Menlo Park, CA
Number of Full-Time Employees: 65
Company Website: www.adverum.com
ABOUT ADVERUM BIOTECHNOLOGIES
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 that includes product candidates to treat wet AMD, A1AT deficiency and hereditary angioedema. Leveraging a next-generation adeno-associated virus (AAV)-based directed evolution platform, the company generates product candidates designed to provide durable efficacy by inducing sustained expression of a therapeutic protein. Adverum has collaboration agreements with Regeneron Pharmaceuticals to research, develop and commercialize gene therapy products for ophthalmic diseases, and Editas Medicine to explore the delivery of genome editing medicines for the treatment of inherited retinal diseases. Adverum has clinical development expertise and core capabilities in vector optimization, process development, manufacturing and assay development.
Susan Washer, President and CEO
Headquarters: Alachua, FL
Number of Full-Time Employees: 65
Company Website: www.agtc.com
February 10, 2017 | AGTC Selected as Top Company in the University of Florida’s 2017 Gator100
February 8, 2017 | AGTC Announces Financial Results and Business Update for the Quarter Ended December 31, 2016
January 24, 2017 | AGTC and Bionic Sight Announce Strategic Collaboration to Develop an Optogenetic Therapy and Device for Visual Deficits and Blindness
AGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients with severe diseases, with an initial focus in ophthalmology. AGTC’s lead product candidates are designed to treat inherited orphan diseases of the eye, caused by mutations in single genes that significantly affect visual function and currently lack effective medical treatments. AGTC’s pipeline includes six ophthalmology development programs across five targets (XLRS, XLRP, achromatopsia, wet age-related macular degeneration and blue cone monochromacy), two non-ophthalmology programs (alpha-1 antitrypsin deficiency and adrenoleukodystrophy) and AGTC is continuing to develop early research studies in additional indications. The company is also exploring genetic defects in cells in the inner ear that lead to deafness and expects to advance several product candidates into development within the next few years.
Jeff Abbey, President and CEO
Headquarters: Durham, NC
Number of Full-Time Employees: 125
Company Website: www.argostherapeutics.com
ABOUT ARGOS THERAPEUTICS
Argos Therapeutics is an immuno-oncology company focused on the development and commercialization of fully personalized immunotherapies for the treatment of cancer using its Arcelis® technology platform. Argos’ most advanced product candidate, AGS-003, is being evaluated in the pivotal ADAPT Phase III clinical trial for the treatment of metastatic renal cell carcinoma (mRCC). The Company is also developing a separate Arcelis®-based product candidate, AGS-004, for the treatment of HIV, which is currently being evaluated in a Phase II clinical trial aimed at HIV eradication in adult patients.
Matt Patterson, President and CEO
Headquarters: San Francisco, CA
Number of Full-Time Employees: 102
Company Website: www.audentestx.com
February 1, 2017 | Audentes Therapeutics Announces FDA Clearance of Investigational New Drug Application for AT342 to Treat Crigler-Najjar Syndrome
January 12, 2017 | Gene Therapy’s Big Surprise May be in an Old South S.F. Warehouse
January 9, 2017 | Audentes Therapeutics Provides Full Year 2017 Strategic Outlook and Financial Guidance
ABOUT AUDENTES THERAPEUTICS
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 products 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.
Geoff MacKay, President and CEO
Headquarters: Cambridge, MA
Number of Full-Time Employees: 9
Company Website: www.avrobio.com
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. AVROBIO is headquartered in Cambridge, MA and has offices in Toronto, ON.
Jeffrey Walsh, Chief Financial and Strategy Officer
Headquarters: Cambridge, MA
Number of Full-Time Employees: 400
Company Website: www.bluebirdbio.com
February 3, 2017 | bluebird bio Announces First Patient Treated with LentiGlobinTM Drug Product Under Amended Study Protocol in HGB-206 Phase 1 Study of Patients with Severe Sickle Cell Disease
ABOUT BLUEBIRD BIO
bluebird bio is leading the gene therapy revolution. The company’s integrated product platforms encompass gene therapy, cancer immunotherapy and gene editing – providing bluebird bio with the potential to treat, and hopefully cure, a broad range of serious diseases. The company’s objective is to develop and bring to market the most advanced products based on the transformative potential of gene therapy as a one-time treatment option for patients with severe genetic and rare diseases and cancer. At the core of this effort is bluebird bio’s collective determination to provide these patients hope for a better life in the face of limited or no long-term safe and effective treatment options.
Thomas Lienard, CEO
Headquarters: Gosselies, Belgium
Number of Full-Time Employees: 110
Company Website: www.bonetherapeutics.com
January 17, 2017 | Bone Therapeutics Provides Business Update for Q4 2016
January 6, 2017 | Bone Therapeutics Granted EUR 2.3 Million in Non-Dilutive Funding by the Walloon Region
ABOUT BONE THERAPEUTICS
Bone Therapeutics is a biotechnology company with a unique approach to the development of cell therapy products for bone fracture repair and fracture prevention. Today, Bone Therapeutics has two first-in-class products which target six indications and offer the potential for additional product extensions: PREOB®, an autologous osteoblastic cell therapy product currently in two pivotal Phase IIB/III clinical trials in Europe for the treatment of osteonecrosis and non-union fractures, as well as in a Phase IIA for severe osteoporosis (treatment-resistant osteoporosis); and ALLOB®, an allogeneic (“off-the-shelf”) osteoblastic cell therapy product currently in three Phase I/IIA proof-of-concept clinical trials for the treatment of delayed-union fractures, spinal fusion procedures and revision spinal fusion. In addition, Bone Therapeutics is conducting preclinical research on next generation products such as combined cell-matrix products for large bone defects and maxillofacial applications or enhanced viscosupplementation for osteoarthritis.
Christian Homsy, M.D., CEO
Headquarters: Mont-Saint-Guibert, Belgium
Number of Full-Time Employees: 90
Company Website: www.celyad.com
February 16, 2017 | Celyad Registers First Hematological Patient in CAR-T NKR-2 THINK Trial
February 6, 2017 | Celyad retains Piper Jaffray & Co. to explore strategic alternatives relating to its C-Cure® cardiovascular assets
January 11, 2017 | Celyad Announces Registration of the First Pancreatic Cancer Patient in its CAR-T NKR-2 THINK Trial in Belgium
Celyad is a clinical-stage biopharmaceutical company focused on the development of specialized cell-based therapies to target severe diseases with significant unmet need, including cancer. Celyad’s Natural Killer Receptor based T-Cell (NKR-T) platform has the potential to treat a broad range of solid and liquid tumors. The company’s lead oncology candidate, the CAR-T NKR-2, has been evaluated in a single dose escalation Phase Ia clinical trial to assess the safety and feasibility of CAR-T NKR-2 cells in patients suffering from AML or MM. THINK, a multiple dose escalation Phase Ib trial, has been initiated to evaluate CAR-T NKR-2 cells in seven indications (AML, MM, colorectal, bladder, ovarian, breast and pancreatic cancers). Celyad was founded in 2007 and is based in Belgium and Boston, MA. Celyad’s ordinary shares are listed on the Euronext Brussels and Paris exchanges, and its American Depository Shares are listed on the NASDAQ Global Market, all under the ticker symbol CYAD.
Headquarters: San Diego, CA
Number of Full-Time Employees: 65
Company Website: www.fatetherapeutics.com
ABOUT FATE THERAPEUTICS
Fate Therapeutics is a 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 cell lines, 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.
John Maslowski, CEO
Headquarters: Exton, PA
Number of Full-Time Employees: 23
Company Website: www.fibrocell.com
January 5, 2017 | Fibrocell Announces FDA Fast Track Designation of FCX-007 for Treatment of Recessive Dystrophic Epidermolysis Bullosa
Fibrocell Science 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, has begun a Phase I/II trial for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). Fibrocell is in preclinical development of FCX-013, its product candidate for the treatment of linear scleroderma. In addition, Fibrocell has a third program in the research phase for the treatment of arthritis and related conditions. Fibrocell’s gene-therapy portfolio is being developed in collaboration with Intrexon Corporation, a leader in synthetic biology.
Bernard Gilly, Ph.D., CEO
(EURONEXT PARIS: SIGHT)
Headquarters: Paris, France
Number of Full-Time Employees: 28
Company Website: www.gensight-biologics.com
January 31, 2017 | GenSight Biologics Receives FDA Orphan Drug Designation for GS030 in Retinitis Pigmentos
ABOUT GENSIGHT BIOLOGICS
GenSight Biologics is a clinical-stage biotechnology company discovering and developing novel therapies for neurodegenerative retinal diseases and diseases of the central nervous system. GenSight Biologics’ pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics for retinitis pigmentosa, to help preserve or restore vision in patients suffering from severe degenerative retinal diseases. GenSight Biologics’ lead product candidate, GS010, is in Phase III trials in Leber’s Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible low vision and legal blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics’ product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.
Adam Gridley, President and CEO
Headquarters: Waltham, MA
Number of Full-Time Employees: 45
Company Website: www.histogenics.com
Histogenics is a leading regenerative medicine company developing and commercializing products in the musculoskeletal segment of the marketplace. Histogenics’ regenerative medicine platform combines expertise in cell processing, scaffolding, tissue engineering, bioadhesives and growth factors to provide solutions to treat musculoskeletal-related conditions. Histogenics’ first investigational product candidate, NeoCart, is currently in Phase III clinical development. NeoCart is an autologous cell therapy designed to treat cartilage defects in the knee using the patient’s own cells. Knee cartilage defects represent a significant opportunity in the United States, with an estimated 500,000 or more applicable procedures each year. 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, characteristics not exhibited in other current treatment options. Histogenics is also developing next-generation allogeneic therapies with Intrexon Corporation.
Rahul Aras, Ph.D., President and CEO
Headquarters: Cleveland, OH
Number of Full-Time Employees: 6
Company Website: www.juventasinc.com
ABOUT JUVENTAS THERAPEUTICS
Juventas Therapeutics is a private, clinical-stage biotechnology company developing novel non-viral gene therapies that activate natural processes to repair the body. Juventas’ lead 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. Juventas is currently enrolling a Phase IIb study in patients with advanced peripheral artery disease.
Arthur Lahr, CEO
Headquarters: Amsterdam, The Netherlands
Number of Full-Time Employees: 42
Company Website: www.kiadis.com
February 6, 2017 | Kiadis Pharma issues clinical and regulatory progress update on ATIR101™ and ATIR201™
ABOUT KIADIS PHARMA
Kiadis Pharma is a clinical-stage biopharmaceutical company focused on research, development and future commercialization of innovative cell-based immunotherapy products that provide for a safer and more
efficacious treatment of blood cancers and inherited blood disorders, improving survival and quality of life and addressing significant unmet medical needs. The company believes that its products address current risks and limitations of allogeneic hematopoietic stem cell transplantation (HSCT). HSCT is considered the most effective curative approach to blood cancers and certain inherited blood disorders, and Kiadis Pharma expects that HSCT will become a treatment option for
many more patients once current risks and limitations are addressed with its products.
Silviu Itescu, Chief Executive
(NASDAQ: MESO | ASX: MSB)
Headquarters: Melbourne, Australia
Number of Full-Time Employees: 80
Company Website: www.mesoblast.com
February 16, 2017 | Positive Nine Month RA Results For Mesoblast Cell Therapy
February 13, 2017 | Published Results Show MSB Cells Effective In RA Model
January 31, 2017 | Mesoblast Director Recognized for Cardiovascular Leadership
Mesoblast is committed to bringing to market its disruptive cellular medicines to treat serious and life-threatening illnesses where there are currently no alternative treatments. Mesoblast has leveraged the company’s proprietary technology platform, which is based on specialized cells known as allogeneic mesenchymal lineage adult stem cells (MLCs), to establish a broad portfolio of late-stage product candidates. These product candidates target advanced stages of diseases with high, unmet medical needs. Mesoblast has three Tier 1 product candidates in Phase III studies and one being evaluated in a Phase II trial. These are:
• MPC-150-IM for advanced chronic and end-stage heart failure
• MPC-06-ID for chronic low back pain due to disc degeneration
• MSC-100-IV for steroid-refractory acute graft versus host disease, and;
• MPC-300-IV for biologic refractory rheumatoid arthritis.
Mesoblast also has a strong pipeline of product candidates for follow-on indications.
Adrien Lemoine, VP, Business Development and Operations
Headquarters: London, UK
Number of Full-Time Employees: 25
Company Website: www.orchard-tx.com
January 9, 2017 | Orchard Therapeutics Ltd. Announces a Manufacturing Alliance with PharmaCell B.V.
ABOUT ORCHARD THERAPEUTICS
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 programmes will use the potential of ex-vivo autologous haematopoietic 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 centres, 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” FierceBiotech company.
Karine Kleinhaus, M.D., Divisional VP, North America
Headquarters: Haifa, Israel
Number of Full-Time Employees: 170
Company Website: www.pluristem.com
January 25, 2017 | Pluristem Announces Closing of Bought Offering and Underwriter’s Exercise in Full of Over-Allotment Option
January 17, 2017 | Pluristem Receives Clearance from Germany to Initiate its Multinational Phase III Trial in Critical Limb Ischemia Targeting Early Conditional Marketing Approval
January 12, 2017 | Pluristem Completes Patient Enrollment in a Large Multinational Phase II Trial in Intermittent Claudication
ABOUT PLURISTEM THERAPEUTICS
Pluristem Therapeutics is a leading developer of placenta-based cell therapy products entering pivotal trials in 2017. Pluristem has reported robust clinical trial data in multiple indications for its patented PLX (PLacental eXpanded) cells. The cell products release a range of therapeutic proteins in response to inflammation, ischemia, hematological disorders and radiation damage. PLX cell products are grown using the company’s proprietary three-dimensional expansion technology. They are off-the-shelf, requiring no tissue matching prior to administration. Pluristem has a strong intellectual property position; company-owned and operated GMP-certified manufacturing and research facilities; strategic partnerships; relationships with major research institutions; and a seasoned management team.
Abid Ansari, VP, Head of Finance
Headquarters: Durham, NC
Number of Full-Time Employees: 58
Company Website: www.precisionbiosciences.com
ABOUT PRECISION BIOSCIENCES
Precision Biosciences is a next generation genome editing company dedicated to improving life. At Precision, the company utilizes a proprietary genome editing method called ARCUS combined with a team made up of some of the leading minds and pioneers in genome editing in an effort to overcome cancers, cure genetic diseases and enable the development of safer, more productive food sources. The backbone of the ARCUS technology is the ARC Nuclease – a fully synthetic enzyme that is very similar to a homing endonuclease but is modified to be a better starting point for the development of new gene editing reagents. ARC Nuclease shares many of the positive attributes of a homing endonuclease, such as small size and unparalleled sequence specificity, but it can more easily be evolved into a custom gene editing tool that recognizes a DNA sequence of the company’s choosing.
Olav Hellebo, CEO
Headquarters: Pencoed, Wales, UK
Number of Full-Time Employees: 60
Company Website: www.reneuron.com
February 15, 2017 | ReNeuron featured in BBC documentary “Andrew Marr: My Brain and Me”
ReNeuron is a leading, clinical-stage cell therapy development company. Based in the UK, 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. ReNeuron’s shares are traded on the London AIM market under the symbol RENE.L
Sandy Macrae, Ph.D., President and CEO
Headquarters: Point Richmond, CA
Number of Full-Time Employees: 140
Company Website: www.sangamo.com
January 11, 2017 | Sangamo Therapeutics Receives Orphan Drug Designation from the FDA for SB-318 Genome Editing Treatment for MPS I
January 6, 2017 | Sangamo BioSciences Changes Name To Sangamo Therapeutics, Underscoring Focus On Clinical Development Of Genomic Therapies
January 5, 2017 | Sangamo BioSciences Announces FDA Clearance Of Investigational New Drug Application For SB-525 Gene Therapy Program For Hemophilia A
ABOUT SANGAMO THERAPEUTICS
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’s proprietary zinc finger nuclease (ZFN) in vivo genome editing approach is being evaluated in Phase I/II clinical trials to treat hemophilia B and lysosomal storage disorders MPS I and MPS II. In 2017 Sangamo is also conducting a Phase I/II clinical trial to evaluate its AAV cDNA human Factor 8 gene therapy approach, SB-525, to treat hemophilia A. Sangamo has a strategic collaboration 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, Sangamo has clinical programs in HIV/AIDS (SB-728). It has established strategic partnerships with companies in non-therapeutic applications of its technology, including Sigma-Aldrich Corporation and Dow AgroSciences.
Robert Millman, CEO
Headquarters: Cambridge, MA
Number of Full-Time Employees: 33
Company Website: www.semma-tx.com
ABOUT SEMMA THERAPEUTICS
Semma Therapeutics was founded to develop transformative therapies for patients who currently depend on insulin injections. Recent 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 the laboratory. These cells develop in islet-like clusters grown from stem cells. Initial preclinical work in animal models of diabetes has shown that transplantation of these cells are sufficient to control blood glucose levels. This breakthrough technology has been exclusively licensed to Semma Therapeutics for the development of a cell-based therapy for diabetes.
David Venables, Ph.D., CEO
Headquarters: Edinburgh, UK
Number of Full-Time Employees: 20
Company Website: www.synpromics.com
January 4, 2017 | Synpromics Announces a Research Collaboration with GE Healthcare to Extend the Toolbox for Production of Biologics
Synpromics enables precision in gene control through the creation of synthetic promoters. The company designs and licenses out customised promoters, for use in cell and gene therapy, based on: expression level, tissue specificity, inducibility, size, kill-switch, environmental and pathologic response. Synpromics has developed the company’s PromPT™ proprietary technology engine to rapidly design and develop novel promoter systems. The platform comprises two major components: (A) Bioinformatics and Machine Learning: The PromPT™ platform facilitates the discovery of endogenous promoter parts from a target genome that can then be used to build novel synthetic promoter candidates. (B) Next Generation Screening: To facilitate the screening of complex libraries containing multitudes of promoter combinations Synpromics has developed a High Content Multiplex (HCM) screening approach. The HCM screen allows complex libraries to be screened: (i) in vivo in the chosen animal model; (ii) in the context of the chosen delivery vector; (iii) with the relevant gene of interest.
Eduardo Bravo, CEO
(EURONEXT BRUSSELS: TIG)
Headquarters: Leuven, Belgium
Number of Full-Time Employees: 70
Company Website: www.tigenix.com
February 17, 2017 | Takeda and TiGenix Report New Data Highlighting Maintenance of Long-Term Remission of Complex Perianal Fistulas in Crohn’s Disease Patients with Cx601
January 31, 2017 | TiGenix Enrolls First Patient in Phase Ib/IIa Clinical Trial of Cx611 in Severe Sepsis
TiGenix is an advanced biopharmaceutical company focused on developing and commercializing novel therapeutics from its proprietary platforms of allogeneic, expanded stem cells. The company’s lead product is Cx601, in registration with the European Medicines Agency for the treatment of complex perianal fistulas in Crohn’s disease patients. TiGenix’s adipose-derived stem cell product candidate Cx611 has completed a Phase I sepsis challenge trial and a Phase I/II trial in rheumatoid arthritis. In 2015, TiGenix acquired Coretherapix, whose lead cellular product candidate, AlloCSC-01, which is currently in a Phase II clinical trial in Acute Myocardial Infarction (AMI). An additional product candidate from the cardiac stem cell-based platform is being developed in a chronic indication. In 2016, TiGenix signed a licensing agreement with Takeda, under which Takeda acquired the exclusive right to commercialize Cx601 for complex perianal fistulas ex-US. TiGenix is headquartered in Belgium and has operations in Spain.
Stephane Boissel, CEO
Headquarters: Valbonne, France
Number of Full-Time Employees: 45
Company Website: www.txcell.com
TxCell is a biotechnology company that develops platforms for innovative, personalized T cell immunotherapies for the treatment of severe chronic inflammatory and autoimmune diseases with high unmet medical need. TxCell is targeting a range of autoimmune diseases (both T cell and B cell-mediated) including Crohn’s disease, lupus nephritis, bullous pemphigoid and multiple sclerosis, as well as transplantation-related inflammatory disorders. TxCell is the only clinical-stage cellular therapy company fully dedicated to the science of regulatory T lymphocytes (Tregs). Tregs are a recently discovered T cell population for which anti-inflammatory properties have been demonstrated. TxCell is developing two proprietary technology platforms, ASTrIA, which is composed of non-modified naturally antigen-specific Tregs, and ENTrIA, which is composed of genetically-engineered Tregs.
Nick Colangelo, President and CEO
Headquarters: Cambridge, MA
Number of Full-Time Employees: 200
Company Website: www.vcel.com
February 1, 2017 | Vericel Announces First MACI Implant in the United States for the Treatment of Symptomatic Cartilage Defects of the Knee
Vericel develops, manufactures and markets expanded autologous cell therapies for the treatment of patients with serious diseases and conditions. The company currently markets two cell therapy products in the United States. Carticel® (autologous cultured chondrocytes) is an autologous chondrocyte implant for the treatment of cartilage defects in the knee in patients who have had an inadequate response to a prior arthroscopic or other surgical repair procedure. Epicel® (cultured epidermal autografts) 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 marketing MACI® (autologous cultured chondrocytes on porcine collagen membrane), an autologous cellularized scaffold product indicated for the repair of symptomatic, single or multiple full-thickness cartilage defects of the knee with or without bone involvement in adults, which has just been approved by the FDA.
Jane Henderson, SVP and Chief Financial Officer
Headquarters: Cambridge, MA
Number of Full-Time Employees: 89
Company Website: www.voyagertherapeutics.com
February 13, 2017 | Voyager Therapeutics Announces Lead Clinical Candidate Selection for Monogenic Form of Amyotrophic Lateral Sclerosis (ALS)
January 25, 2017 | Voyager Therapeutics Announces Publication of Vectored Anti-Tau Monoclonal Antibody Study in The Journal of Neuroscience
January 20, 2017 | Voyager Therapeutics Announces Updates from Phase 1b Trial of VY-AADC01 for Advanced Parkinson’s Disease
ABOUT VOYAGER THERAPEUTICS
Voyager Therapeutics is a clinical-stage gene therapy company developing life-changing treatments for severe diseases of the central nervous system (CNS). Gene therapy has the potential to transform the treatment of CNS and other diseases by providing a one-time therapy that dramatically improves patients’ lives. Voyager is committed to advancing the field of adeno-associated virus (AAV) gene therapy through innovation and investment in vector optimization and engineering, dosing techniques as well as process development and production.