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
March 8, 2017 | Abeona Therapeutics Receives Orphan Drug Designation in the European Union for EB-101 Gene Therapy Clinical Trial for Epidermolysis Bullosa
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
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
March 22, 2017 | AVROBIO, Inc. Expands Rare Disease Pipeline with Gene Therapy to Treat Gaucher Disease
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.
Peter Altman, Ph.D., CEO
Headquarters: San Carlos, CA
Number of Full-Time Employees: 20
Company Website: www.biocardia.com
February 28, 2017 | Johns Hopkins Medicine, Maryland Stem Cell Research Fund and BioCardia Announce First Patient Treated with CardiAMP Cell Therapy for Ischemic Heart Failure in Phase III Clinical Trial
BioCardia is a clinical-stage regenerative medicine company developing proprietary comprehensive biotherapeutic solutions for cardiovascular disease in its CardiAMP™ and CardiALLO™ therapies. Clinical results supporting both therapeutic programs are compelling and build upon the most rigorous data in the field to-date with intramyocardial delivery of marrow-derived cells. These programs are enabled by the company’s Helix™ transendocardial delivery systems and Morph® vascular access products, which are partnered to enable other promising biotherapeutic programs.
Jeffrey Walsh, Chief Financial and Strategy Officer
Headquarters: Cambridge, MA
Number of Full-Time Employees: 400
Company Website: www.bluebirdbio.com
March 1, 2017 | bluebird bio Announces Publication of Case Study on First Patient with Severe Sickle Cell Disease Treated with Gene Therapy in The New England Journal of Medicine
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
March 9, 2017 | Bone Therapeutics Completes Recruitment of 16 Patients in ALLOB® Phase I/IIA Delayed-union Study
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.
David Mazzo, Ph.D., CEO
Headquarters: Basking Ridge, NJ
Number of Full-Time Employees: 221
Company Website: www.caladrius.com
February 23, 2017 | Caladrius Biosciences Awarded $12.2 Million Grant from CIRM in Support of Phase 2 Clinical Trial of CLBS03 to Treat Type 1 Diabetes
January 25, 2017 | Caladrius Biosciences Announces Addition of Three Clinical Sites, Including University of California, San Francisco, for the Ongoing Phase 2 Study of CLBS03 in T1D
ABOUT CALADRIUS BIOSCIENCES
Caladrius Biosciences is a cell therapy company with cell therapy products in development based on multiple technology platforms and targeting autoimmune and cardiology indications. The company’s subsidiary, PCT, is a leading development and manufacturing partner exclusively focused on the cell therapy industry and has served over 100 clients since 1999. PCT provides a wide range of innovative services including product and process development, GMP manufacturing, engineering and automation, cell and tissue processing, logistics, storage and distribution, as well as expert consulting and regulatory support.
Linda Marban, Ph.D., CEO
Headquarters: Beverly Hills, CA
Number of Full-Time Employees: 40
Company Website: www.capricor.com
ABOUT CAPRICOR THERAPEUTICS
Capricor Therapeutics is a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class biological therapies for the treatment of cardiac and other medical conditions. Capricor’s lead candidate, CAP-1002, is a cardiac cell therapy that is currently being evaluated for the treatment of heart disease associated with Duchenne muscular dystrophy and myocardial infarction (heart attack). Capricor is exploring the use of CAP-2003, its exosome product candidate, in various therapeutic areas including the treatment of ophthalmic disorders.
Christian Homsy, M.D., CEO
Headquarters: Mont-Saint-Guibert, Belgium
Number of Full-Time Employees: 90
Company Website: www.celyad.com
March 8, 2017 | Celyad Obtains FDA Approval to Initiate the NKR-2 CAR T Cells THINK Trial in the USA
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
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.
Scott Wolchko, President and CEO
Headquarters: San Diego, CA
Number of Full-Time Employees: 65
Company Website: www.fatetherapeutics.com
March 13, 2017 | Fate Therapeutics Announces FDA Clearance of Investigational New Drug Application for FATE-NK100 Natural Killer Cell Cancer Immunotherapy
February 27, 2017 | Fate Therapeutics and Regents of the University of Minnesota Expand Research Collaboration for Clinical Translation of Engineered iPSC-Derived Natural Killer Cell Cancer Immunotherapy
January 5, 2017 | Fate Therapeutics Announces First Patient Treated in ProTmune™ PROTECT Clinical Trial for the Prevention of Graft-Versus-Host Disease
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
March 9, 2017 | Histogenics Announces Publication of Biomechanical and Structural Data From Human Engineered Cartilage Testing
January 11, 2017 | Histogenics Announces Publication of MRI Data From NeoCart® Phase 1 and Phase 2 Clinical Trials in the American Journal of Sports Medicine
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.
Karen Aiach, CEO
(EURONEXT PARIS: LYS)
Headquarters: Neuilly-sur-Seine, France
Number of Full-Time Employees: 15
Company Website: www.lysogene.com
February 21, 2017 | Lysogene Receives Orphan Drug Designation from EMA for LYS-GM101 for Treatment of GM1 Gangliosidosis
February 2, 2017 | Lysogene Receives Orphan Drug Designation from FDA for LYS-GM101 for treatment of GM1 Gangliosidosis
January 18, 2017 | Lysogene Receives Rare Pediatric Disease Designation from FDA for LYS-GM101 for Treatment of GM1 Gangliosidosis
Lysogene is a global biotechnology company, a leader in the basic research and clinical development of gene therapy for neurodegenerative disorders. Lysogene’s mission is to radically improve the health of patients suffering from incurable life threatening conditions by developing AAV vectors that have demonstrated their effectiveness in safely delivering genetic material to the central nervous system. Lysogene’s most advanced product candidate is rAAV vector serotype rh.10 carrying the human N-sulfoglucosamine sulfohydrolase (hSGSH) for the treatment of mucopolysaccharidosis IIIA (MPS IIIA). The recently completed Phase I/II study in four MPS IIIA children demonstrated that the gene therapy and neurosurgical procedure is safe, well tolerated and exploratory efficacy profiles are encouraging (Tardieu 2014). A multi-national Phase IIb pivotal clinical trial is under preparation with a second generation gene therapy. Lysogene also has a program underway for the development of a rAAVrh.10 carrying the human beta-galactosidase for the treatment of GM1 gangliosidosis.
Silviu Itescu, Chief Executive
(NASDAQ: MESO | ASX: MSB)
Headquarters: Melbourne, Australia
Number of Full-Time Employees: 80
Company Website: www.mesoblast.com
March 7, 2017 | FDA Grants Mesoblast Fast Track Designation For GVHD
February 16, 2017 | Positive Nine Month RA Results For Mesoblast Cell Therapy
February 13, 2017 | Published Results Show MSB Cells Effective In RA Model
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.
John Dawson, CEO
Headquarters: Oxford, UK
Number of Full-Time Employees: 220
Company Website: www.oxfordbiomedica.co.uk
ABOUT OXFORD BIOMEDICA
The combined strength of Oxford BioMedica’s in-house development know-how, bioprocessing facilities and intellectual property creates a uniquely powerful integrated platform, positioning the company at the forefront of gene and cell therapy. The company believes that gene therapy will become a mainstay of patient therapy in the future. Oxford BioMedica’s long-term goal is to become an integrated, self-financing gene and cell therapy business using the company’s LentiVector® platform capabilities to continue investing in Oxford BioMedica’s early-stage and preclinical pipeline to generate further valuable new product candidates. The company will also continue to invest in lentiviral vector technology to ensure that the Group retains its leadership position, which will help maximize bioprocessing revenues.
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
March 1, 2017 | Sangamo Therapeutics Receives Orphan Drug Designation from the FDA for SB-913 Genome Editing Treatment for MPS II
February 27, 2017 | Sangamo Therapeutics Receives Rare Pediatric Disease Designation From FDA For SB-318 In Vivo Genome Editing Therapeutic For MPS I
January 11, 2017 | Sangamo Therapeutics Receives Orphan Drug Designation from the FDA for SB-318 Genome Editing Treatment for MPS I
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
March 13, 2017 | TiGenix Announces Top-Line Phase I/II Results of AlloCSC-01 in Acute Myocardial Infarction
March 9, 2017 | TiGenix Announces Positive Topline Week-104 Data for Cx601 ADMIRE-CD Trial
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
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
(EURONEXT PARIS: TXCL)
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.
Matt Kapusta, CEO
Headquarters: Lexington, MA
Number of Full-Time Employees: 200
Company Website: www.uniqure.com
January 30, 2017 | uniQure Announces FDA Breakthrough Therapy Designation for AMT-060 in Hemophilia B
January 25, 2017 | uniQure Appoints Alexander Kuta as Senior Vice President of Regulatory Affairs
uniQure is delivering on the promise of gene therapy, single treatments with potentially curative results. uniQure developed a modular 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 has 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 is developed both internally and through multiple collaborations using the company’s innovative, modular technology platform, including proprietary commercial-grade industry-leading manufacturing capabilities in two geographical locations. Through the company’s collaborations and strategic partnership with Bristol-Myers Squibb to develop gene therapies for cardiovascular diseases, uniQure has made the next step towards developing gene therapies targeting chronic and degenerative diseases that affect larger populations.
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.