Aduro Biotech (ADRO)
An immunotherapy company, focuses on the discovery, development, and commercialization of therapies to harness the body's natural immune system for the treatment of patients with challenging diseases. The company is developing ADU-S100, which is in Phase 1 dose escalation clinical trial and Phase 1b dose escalation clinical trial in combination with spartalizumab to treat patients with advanced, metastatic treatment-refractory solid tumors; Phase I clinical trial in combination with ipilimumab for the treatment of relapsed and refractory melanoma; and Phase II clinical trial in combination with pembrolizumab, an approved anti-PD-1 antibody in patients with squamous cell carcinoma of the head and neck. It is also developing product candidates to prevent or control immune responses through the stimulator of interferon genes pathway. In addition, the company is developing BION-1301, a fully blocking humanized monoclonal anti-APRIL antibody that is in Phase 1 clinical trial for IgA nephropathy; and CD27, a co-stimulatory receptor, which is in preclinical studies expressed on different immune cells, such as T-lymphocytes and natural killer cells. The company was formerly known as Oncologic, Inc. and changed its name to Aduro Biotech, Inc. in June 2008. Aduro Biotech, Inc. was founded in 2000 and is headquartered in Berkeley, California.
Collaboration agreements with Novartis Pharmaceuticals Corporation, Eli Lilly and Company, and Merck Sharp and Dohme B.V.; and license agreements with Karagen Pharmaceuticals, Inc., UC Berkeley, and Memorial Sloan Kettering Cancer Center.
Adverum Biotechnologies (ADVM)
A clinical-stage gene therapy company, engages in developing gene therapy product candidates to treat ocular and rare diseases. Its pipeline of product candidates includes ADVM-022, an adeno- associated virus (AAV). 7m8-aflibercept for the treatment of wet age-related macular degeneration and diabetic retinopathy; ADVM-043, an investigational gene therapy candidate for the treatment of alpha-1 antitrypsin deficiency; and ADVM-053, a preclinical gene therapy product candidate for the treatment of hereditary angioedema. The company was formerly known as Avalanche Biotechnologies, Inc. and changed its name to Adverum Biotechnologies, Inc. in May 2016. Adverum Biotechnologies, Inc. was founded in 2006 and is headquartered in Redwood City, California.
The company has collaboration agreements with Editas Medicine, Inc. to leverage its proprietary AAV vectors for genome editing technologies to treat up to five ophthalmic indications; Regeneron Pharmaceuticals, Inc.; and GenSight Biologics S.A. to develop gene therapy products to deliver certain therapeutic transgenes.
Applied Genetic Technologies (AGTC)
A clinical-stage biotechnology company, develops transformational genetic therapies for patients suffering from rare and debilitating diseases. The company's advanced product candidates consist of three ophthalmology development programs across two targets, including X-linked retinitis pigmentosa, which is in Phase I/II clinical trials; and achromatopsia that is in Phase I/II clinical trials. The company also has initiated one preclinical program in otology and three preclinical programs in targeting central nervous system disorders, including one in adrenoleukodystrophy. The company was founded in 1999 and is headquartered in Alachua, Florida.
Collaboration agreements with Synpromics Limited and University of Florida; Bionic Sight, LLC; and Otonomy, Inc.
Alnylam Pharmaceuticals (ALNY)
A biopharmaceutical company, focuses on discovering, developing, and commercializing RNA interference (RNAi) therapeutics. The company's pipeline of investigational RNAi therapeutics focus on genetic medicines, cardio-metabolic diseases, hepatic infectious diseases, and central nervous system/ocular diseases. Its marketed products include ONPATTRO (patisiran), a lipid complex injection for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults; and GIVLAARI for the treatment of adults with acute hepatic porphyria (AHP). In addition, the company is developing givosiran for the treatment of patients with AHP; lumasiran for the treatment of primary hyperoxaluria type 1, or PH1; patisiran for the treatment of transthyretin amyloidosis, or ATTR amyloidosis, with cardiomyopathy; and vutrisiran for the treatment of ATTR amyloidosis. The company was founded in 2002 and is headquartered in Cambridge, Massachusetts.
Alnylam Pharmaceuticals, Inc. has strategic alliances primarily with Sanofi Genzyme; The Medicines Company; Ionis Pharmaceuticals, Inc.; and Regeneron Pharmaceuticals, Inc., as well as collaboration with Vir Biotechnology, Inc. to advance RNAi therapeutics for the treatment of coronavirus infection, including COVID-19. It also has a collaboration and license agreement with Dicerna Pharmaceuticals, Inc. to develop and commercialize investigational ribonucleic acid interference therapeutics for the treatment of alpha-1 antitrypsin deficiency-associated liver disease, as well as Vir Biotechnology, Inc. to develop and commercialize RNAi therapeutics.
Athersys (ATHX)
A biotechnology company, focuses on the research and development activities in the field of regenerative medicine. Its clinical development programs are focused on treating neurological conditions, cardiovascular diseases, inflammatory and immune disorders, and pulmonary and other conditions. The company's lead platform product includes MultiStem cell therapy, an allogeneic stem cell product, which is in Phase III clinical study for the treatment of patients suffering from neurological damage from an ischemic stroke, as well as in Phase II clinical study for the treatment of patients with acute myocardial infarction, and has completed Phase I clinical study for the treatment of patients suffering from leukemia or various other blood-borne cancers. It also develops MultiStem cell therapy to promote tissue repair and healing for animal patients, including those suffering from serious conditions with unmet medical needs. Athersys, Inc. was founded in 1995 and is headquartered in Cleveland, Ohio.
Has license and collaboration agreements with Healios K.K. to develop and commercialize MultiStem cell therapy for ischemic stroke, acute respiratory distress syndrome, and ophthalmological indications, as well as for the treatment of liver, kidney, pancreas, and intestinal tissue diseases; and the University of Minnesota to develop MultiStem cell therapy platform.
AxoGen (AXGN)
Provides surgical solutions for physical damage or transection to peripheral nerves. The company's products include Avance Nerve Graft, a biologically active off-the-shelf processed human nerve allograft for bridging severed nerves without the comorbidities associated with a second surgical site; AxoGuard Nerve Connector, a porcine submucosa extracellular matrix (ECM) coaptation aid for tensionless repair of severed peripheral nerves; and AxoGuard Nerve Protector, a porcine submucosa ECM product that is used to wrap and protect damaged peripheral nerves, as well as reinforces the nerve reconstruction while preventing soft tissue attachments. Its products also comprise Axoguard Nerve Cap, a porcine submucosa ECM product that is used to protect a peripheral nerve end, as well as separates the nerve from the surrounding environment to reduce the development of symptomatic or painful neuroma; and Avive Soft Tissue Membrane, a minimally processed human umbilical cord membrane that can be used as a resorbable soft tissue covering to separate tissues in the surgical bed. In addition, the company offers AxoTouch two-point discriminator, a tool that is used for measuring the innervation density of surface area of the skin. It provides its products to hospitals, surgery centers, and military hospitals plastic reconstructive surgeons, orthopedic and plastic hand surgeons, and various oral and maxillofacial surgeons in the United States, Canada, the United Kingdom and other European countries, South Korea, and internationally. AxoGen, Inc. is headquartered in Alachua, Florida.
Brainstorm Cell Technologies (BCLI)
http://www.brainstorm-cell.com
Engages in the development and commercialization of central nervous system (CNS) adult stem cell therapies designed to address the unmet medical needs of patients with debilitating neurodegenerative diseases. The company holds rights to develop and commercialize its NurOwn technology that utilizes a patient's own cells, which are engineered outside the body, to produce and secrete factors known to promote neuronal survival. It is developing NurOwn for various neurodegenerative diseases, including its lead indication, which is in Phase III clinical trial for the treatment of amyotrophic lateral sclerosis, as well as in Phase II for the treatment of multiple sclerosis, and preclinical trial for Parkinson's disease, Huntington's disease, and autism spectrum disorder. The company was formerly known as Golden Hand Resources Inc. and changed its name to Brainstorm Cell Therapeutics Inc. in November 2004 to reflect its new line of business in the development of novel cell therapies for neurodegenerative diseases. Brainstorm Cell Therapeutics Inc. was founded in 2000 and is headquartered in New York, New York.
Bellicum Pharmaceuticals (BLCM)
Focuses on discovering and developing novel cellular immunotherapies for the treatment of hematological cancers, solid tumors, and orphan inherited blood disorders in the United States and internationally. The company's clinical product candidates include Rivo-cel that is in Phase II/III clinical trials to improve hematopoietic stem cell transplantation outcomes in the treatment of hematologic malignancies, including leukemias, lymphomas, and inherited blood disorders; and BPX-601, an autologous GoCAR-T product candidate, which is in Phase I/II clinical trials for treating solid tumors expressing the prostate stem cell antigen. Its clinical product candidates also comprise BPX-603, a dual-switch GoCAR-T product candidate to treat solid tumors that express the human epidermal growth factor receptor 2 antigen; BPX-802, a dual switch Go-CAR-T product candidate to treat an antigen expressed in hematological malignancies; and BPX-701, a T cell receptor candidate, which is in Phase I clinical trial for the treatment of malignant cells expressing the preferentially-expressed antigen in melanoma. Bellicum Pharmaceuticals, Inc. was founded in 2004 and is headquartered in Houston, Texas.
Ccollaboration and license agreements with Adaptimmune Therapeutics plc, Ospedale Pediatrico Bambino Gesù, Leiden University Medical Centre, Agensys, Inc., BioVec Pharma, Inc., Leiden University Medical Centre, ARIAD Pharmaceuticals, Inc., and Baylor College of Medicine.
BioLife Solutions (BLFS)
http://www.biolifesolutions.com
Develops, manufactures, and markets grade cell and tissue hypothermic storage and cryopreservation freeze media products for cells, tissues, and organs in the United States. Its products are serum-free and protein-free solutions, which are formulated to reduce preservation-induced cell damage and death. The company offers CryoStor cryopreservation freeze media products to mitigate temperature-induced molecular cell stress responses during freezing and thawing; and HypoThermosol FRS, a hypothermic storage and shipping media product to mitigate temperature-induced molecular cell stress responses that occur during chilling and re-warming of biologics, intermediate products, and final cell products intended for research and clinical applications. It also provides BloodStor freeze media products, such as BloodStor 55-5 and BloodStor 100 to cryopreserve stem and other cells isolated from umbilical cord and peripheral blood, and bone marrow; and cell thawing media, which offers Dextran and saline for washing cryopreserved cells and tissues to dilute or remove cryoprotectants. In addition, the company provides custom product formulation and packaging services. It markets its products to the regenerative medicine, bio-banking, and drug discovery markets, including cell and gene therapy companies, research institutions, hospital-based stem cell transplant centers, cell therapy contract manufacturing organizations, contract development manufacturing organizations, pharmaceutical companies, cord blood and adult stem cell banks, and hair transplant centers, as well as suppliers of cells to the drug discovery, toxicology testing, and diagnostic markets.
Markets and sells its products directly, as well as through third party distributors. BioLife Solutions, Inc. was founded in 1987 and is headquartered in Bothell, Washington.
bluebird bio (BLUE)
Focuses on developing transformative gene therapies for severe genetic diseases and cancer. Its product candidates in severe genetic diseases include LentiGlobin, which is in various clinical studies for the treatment of transfusion-dependent ß-thalassemia and severe sickle cell disease; and Lenti-D that is in Phase II/III clinical trials for the treatment of cerebral adrenoleukodystrophy, a rare hereditary neurological disorder. The company's product candidates in oncology include bb2121 and bb21217, which are chimeric antigen receptor T (CAR T) cell product candidates for the treatment of multiple myeloma. It has a strategic collaboration with Celgene Corporation to discover, develop, and commercialize disease-altering gene therapies in oncology; and Regeneron Pharmaceuticals, Inc. to discover, develop, and commercialize various immune cell therapies for cancer. The company was formerly known as Genetix Pharmaceuticals, Inc., and changed its name to bluebird bio, Inc. in September 2010. bluebird bio, Inc. was founded in 1992 and is headquartered in Cambridge, Massachusetts.
Collaborations with Medigene AG to discover T cell receptor (TCR) product candidates in the field of cancer; Gritstone Oncology, Inc. to discover TCR product candidates in the field of cancer; TC BioPharm Limited to research and develop gamma delta CAR T cells directed at hematologic and solid tumor targets; and Novo Nordisk A/S to develop in vivo genome editing treatments for genetic diseases.
Biostage (BSTG)
Engages in developing bioengineered organ implants based on its Cellframe technology. The company's Cellframe technology comprises a biocompatible scaffold that is seeded with the recipient's own stem cells. It is developing its Cellframe technology to treat life-threatening conditions of the esophagus, bronchus, or trachea. The company was formerly known as Harvard Apparatus Regenerative Technology, Inc. and changed its name to Biostage, Inc. in March 2016. Biostage, Inc. was founded in 2007 and is headquartered in Holliston, Massachusetts.
Caladrius Biosciences (CLBS)
http://www.caladrius.com … develops cellular therapeutic product candidates to address diseases and conditions caused by ischemia. Its product candidates include developmental treatments for cardiovascular diseases, such as CLBS12 for the treatment of critical limb ischemia; CLBS16, which is in Phase II clinical trial for the treatment of coronary microvascular dysfunction; CLBS14, a regenerative medicine advanced therapy for treating no-option refractory disabling angina. The company was formerly known as NeoStem, Inc. and changed its name to Caladrius Biosciences, Inc. in June 2015. Caladrius Biosciences, Inc. was founded in 1980 and is headquartered in Basking Ridge, New Jersey.
Cellectis SA (CLLS)
http://www.cellectis.com … develops immuno-oncology products based on gene-edited T-cells that express chimeric antigen receptors to target and eradicate cancer cells. It operates through two segments, Therapeutics and Plants. The company is developing UCART19, an allogeneic T-cell product candidate for the treatment of CD19-expressing hematologic malignancies, such as acute lymphoblastic leukemia (ALL); ALLO-501 to treat relapsed/refractory diffuse large B-cell lymphoma and follicular lymphoma; UCART123 for the treatment of acute myeloid leukemia (AML); and UCART22 to treat B-cell acute lymphoblastic leukemia. It is also developing UCARTCS1 and ALLO-715 for the treatment of multiple myeloma. In addition, the company produces high oleic soybean oil, other soybean products, and fiber wheat. The company was founded in 1999 and is headquartered in Paris, France.
Strategic alliances with Allogene Therapeutics, Inc.; Les Laboratoires Servier; The University of Texas M.D. Anderson Cancer Center; and Iovance Biotherapeutics.
CRISPR Therapeutics (CRSP)
Develops its products using Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9), a gene editing technology that allows for precise directed changes to genomic DNA. It has a portfolio of therapeutic programs in a range of disease areas, including hemoglobinopathies, oncology, regenerative medicine, and rare diseases. The company's lead product candidate is CTX001, an ex vivo CRISPR gene-edited therapy for treating patients suffering from transfusion-dependent beta thalassemia or severe sickle cell disease in which a patient's hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin in red blood cells. It is also developing CTX110, a donor-derived gene-edited allogeneic CAR-T therapy targeting cluster of differentiation 19 positive malignancies. In addition, the company is developing allogeneic CAR-T programs comprising CTX120 targeting B-cell maturation antigen for the treatment of multiple myeloma; and CTX130 for the treatment of solid tumors and hematologic malignancies. Further, it engages in developing regenerative medicine programs in diabetes; and in vivo and other genetic disease programs to treat glycogen storage disease Ia, Duchenne muscular dystrophy, and cystic fibrosis. CRISPR Therapeutics AG is headquartered in Zug, Switzerland.
Strategic partnerships with Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, and ViaCyte, Inc.
Editas Medicine (EDIT)
Editas Medicine focuses on developing transformative genomic medicines to treat a range of serious diseases. The company develops a proprietary genome editing platform based on CRISPR technology, which includes CRISPR/Cas9, CRISPR/Cas12a, and engineered forms of both of these CRISPR systems to target genetically addressable diseases and therapeutic areas. It also develops EDIT-101, which is in Phase 1/2 clinical trial for the treatment of Leber Congenital Amaurosis type 10, a genetic form of vision loss that leads to blindness in childhood. In addition, the company develops EDIT-102 for the treatment of Usher Syndrome 2A, which is a form of retinitis pigmentosa that also includes hearing loss; autosomal dominant retinitis pigmentosa 4, a progressive form of retinal degeneration; and EDIT-301 treat sickle cell disease and beta-thalassemia. The company was formerly known as Gengine, Inc. and changed its name to Editas Medicine, Inc. in November 2013. Editas Medicine, Inc. was founded in 2013 and is headquartered in Cambridge, Massachusetts.
Research collaboration with Juno Therapeutics, Inc. to develop engineered T cells for cancer; strategic alliance and option agreement with Allergan Pharmaceuticals International Limited to discover, develop, and commercialize new gene editing medicines for a range of ocular disorders; strategic research collaboration Asklepios BioPharmaceutical, Inc. to develop a therapy to treat a neurological disease; and research collaboration with Sandhill Therapeutics, Inc. to develop allogeneic healthy donor derived NK cell medicines for the treatment of solid tumors.
Fate Therapeutics (FATE)
http://www.fatetherapeutics.com
Develops programmed cellular immunotherapies for cancer and immune disorders worldwide. Its NK- and T-cell immuno-oncology programs under development includes FT516 for the treatment of acute myeloid leukemia (AML) and B-cell lymphoma, FT596 to treat B-cell lymphoma and CLL, FT538 to treat AML and multiple myeloma, FT576 to treat multiple myeloma, FT819 to treat B-cell malignancies, and FT-ONO1 to treat hematologic malignancies; and FT500, FT516, and FT-ONO2 for the treatment of advanced solid tumors. The company's NK- and T-cell immuno-oncology programs under development also includes ProTmune for the treatment of hematologic malignancies; FATE-NK100 for the treatment of recurrent ovarian cancer; and FATE-NK100 for the treatment of advanced solid tumors. Fate Therapeutics, Inc. was founded in 2007 and is headquartered in San Diego, California.
Collaboration and option agreement with Ono Pharmaceutical Co. Ltd. for the development and commercialization of two off-the-shelf iPSC-derived CAR T-cell product candidates; strategic research collaboration and license agreement with Juno Therapeutics, Inc. to screen for and identify small molecule modulators that enhance the therapeutic properties of genetically-engineered T-cell immunotherapies; and a collaboration and option agreement with Janssen Biotech, Inc.
Homology Medicine (FIXX)
http://www.homologymedicines.com
Focuses on transforming the lives of patients suffering from rare genetic diseases. Its proprietary platform is designed to utilize its human hematopoietic stem cell derived adeno-associated virus vectors (AAVHSCs) to deliver genetic medicines in vivo either through a gene therapy or nuclease-free gene editing across a range of genetic disorders. The company's various set of AAVHSCs allows company to target, through a single injection, a range of disease-relevant tissues, including the liver, central nervous system, peripheral nervous system, bone marrow, muscle, and eye. Its lead product candidate is HMI-102, which is in Phase 1/2 pheNIX clinical trial, a gene therapy for the treatment of phenylketonuria (PKU) in adults. The company also develops HMI-103 for the treatment of PKU in pediatric patients; and HMI-202 to treat metachromatic leukodystrophy. Homology Medicines, Inc. was founded in 2015 and is based in Bedford, Massachusetts.
Global Blood Therapeutics (GBT)
Engages in the discovery, development, and delivery of treatments for underserved patient communities. It is developing its lead product candidate, Oxbryta (voxelotor) tablets, an oral, once-daily therapy for sickle cell disease (SCD). The company is evaluating Oxbryta that has completed Phase 3 clinical trial in adult and adolescent patients with SCD. It is also evaluating the safety and pharmacokinetics of single and multiple doses of Oxbryta in a Phase 2a clinical trial of adolescent and pediatric patients with SCD. Global Blood Therapeutics, Inc. was founded in 2011 and is headquartered in South San Francisco, California.
License and collaboration agreement with Syros Pharmaceuticals, Inc. to discover, develop, and commercialize therapies for SCD) and beta thalassemia. Global Blood Therapeutics, Inc. was founded in 2011 and is headquartered in South San Francisco, California.
Ionis Pharmaceuticals (IONS)
Discovers and develops RNA-targeted therapeutics in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; and TEGSEDI, an injection for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. It is involved in developing neurology products that include IONIS-HTTRx for Huntington's diseases; and IONIS-SOD1Rx and IONIS-C9Rx for amyotrophic lateral sclerosis and IONIS-MAPTRx for Alzheimer's diseases. The company is also developing severe and rare disease products, such as WAYLIVRA, a treatment for familial chylomicronemia syndrome; WAYLIVRA, a treatment for familial partial lipodystrophy; AKCEA-TTR-LRx for transthyretin amyloidosis and IONIS-GHR-LRx for acromegaly; and IONIS-TMPRSS6-LRx for beta-thalassemia and iron toxicity, IONIS-PKKRx/IONIS-PKK-LRx for hereditary angioedema, and IONIS-ENAC-2.5Rx for cystic fibrosis. In addition, its cardio metabolic and renal drugs include AKCEA-ANGPTL3-LRx for cardio metabolic disorders, IONIS-FXIRx for clotting disorders, AKCEA-APO(a)-LRx and AKCEA-APOCIII-LRx for cardiovascular diseases, and IONIS-DGAT2Rx for nonalcoholic steatohepatitis; IONIS-AGT-LRX for the treatment of resistant hyper tension; and IONIS-FXI-LRx for clotting disorders and IONIS-AZ4-2.5-LRx for cardiovascular diseases. Further, the company develops products for oncology that include IONIS-AR-2.5Rx and danvatirsen for cancers; and IONIS-HBVRx and IONIS-HBV-LRx for hepatitis b virus infection, and IONIS-FB-LRx for compliment meditated disease, and IONIS-JBI1-2.5Rx for gastrointestinal autoimmune diseases. Ionis Pharmaceuticals, Inc. was founded in 1989 and is based in Carlsbad, California.
MiMedx (Pink sheets: MDXG)
Develops and markets regenerative biologics utilizing human placental tissue allografts with patent-protected processes for various sectors of healthcare. It processes the human placental tissue utilizing its proprietary PURION Process to produce allografts. The company's proprietary processing methodology employs aseptic processing techniques in addition to terminal sterilization. Its biomaterial platform technologies include AmnioFix and EpiFix that are tissue technologies processed from human amniotic membrane derived from donated placental tissue for homologous applications; OrthoFlo, an amniotic fluid-derived allograft for homologous applications; Physio, a bone grafting material comprising 100% bone tissue with no added carrier; and CollaFix, a technology platform derived from collagen fiber technology designed to mimic the natural composition, structure, and mechanical properties of musculoskeletal tissues to augment their repair. The company also offers EpiCord, an umbilical cord allograft that provides a connective tissue matrix to replace or supplement damaged or inadequate integumental tissue; AmnioCord, an umbilical cord allograft that offers a protective environment for the healing process; and AmnioFill, a cellular tissue matrix allograft that enhances healing. Its products have applications in the areas of wound care, burns, surgery, orthopedics, spine, sports medicine, ophthalmology, and dentistry. The company sells its products through direct sales force, and independent stocking distributors, and third party representatives in the United States, as well as independent distributors internationally. MiMedx Group, Inc. develops and markets regenerative biologics utilizing human placental tissue allografts with patent-protected processes for various sectors of healthcare. It processes the human placental tissue utilizing its proprietary PURION Process to produce allografts. The company's proprietary processing methodology employs aseptic processing techniques in addition to terminal sterilization. Its biomaterial platform technologies include AmnioFix and EpiFix that are tissue technologies processed from human amniotic membrane derived from donated placental tissue for homologous applications; OrthoFlo, an amniotic fluid-derived allograft for homologous applications; Physio, a bone grafting material comprising 100% bone tissue with no added carrier; and CollaFix, a technology platform derived from collagen fiber technology designed to mimic the natural composition, structure, and mechanical properties of musculoskeletal tissues to augment their repair. The company also offers EpiCord, an umbilical cord allograft that provides a connective tissue matrix to replace or supplement damaged or inadequate integumental tissue; AmnioCord, an umbilical cord allograft that offers a protective environment for the healing process; and AmnioFill, a cellular tissue matrix allograft that enhances healing. Its products have applications in the areas of wound care, burns, surgery, orthopedics, spine, sports medicine, ophthalmology, and dentistry. The company sells its products through direct sales force, and independent stocking distributors, and third-party representatives in the United States, as well as independent distributors internationally. MiMedx Group, Inc. is headquartered in Marietta, Georgia.
Mesoblast (MESO)
Develops and commercializes allogeneic cellular medicines. The company offers products in the areas of cardiovascular, spine orthopedic disorder, oncology, hematology, and immune-mediated and inflammatory diseases. Its proprietary regenerative medicine technology platform is based on specialized cells known as mesenchymal lineage adult stem cells. The company's products under the Phase III clinical trials include MSC-100-IV for steroid refractory acute graft versus host disease; MPC-150-IM for advanced heart failure; and MPC-06-ID for chronic low back pain due to degenerative disc disease. It is also developing MPC-300-IV that is in Phase II clinical trials for the treatment of biologic refractory rheumatoid arthritis, diabetic kidney diseases, and type 2 diabetic nephropathy. It operates in the United States, Australia, Singapore, the United Kingdom, and Switzerland. The company was founded in 2004 and is headquartered in Melbourne, Australia.
Strategic partnerships with Tasly Pharmaceutical Group to offer MPC-150-IM for heart failure and MPC-25-IC for heart attacks in China; JCR Pharmaceuticals Co. Ltd. for the treatment of wound healing in patients with epidermolysis bullosa; and Grünenthal to develop and commercialize cell therapy for the treatment of chronic low back pain.
Intellia Therapeutics (NTLA)
A genome editing company, focuses on the development of therapeutics. It utilizes a biological tool known as the Clustered, Regularly Interspaced Short Palindromic Repeats/CRISPR associated 9 (CRISPR/Cas9) system. The company develops in vivo programs focusing on liver diseases, including transthyretin amyloidosis and hereditary angioedema, as well as other research programs comprising primary hyperoxaluria Type 1, alpha-1 antitrypsin deficiency, and Hemophilia B. Its ex vivo pipeline includes proprietary programs focused on developing engineered cell therapies to treat various oncological and autoimmune diseases; partnered programs focused on chimeric antigen receptor T cells and hematopoietic stem cells; and acute myeloid leukemia. The company was formerly known as AZRN, Inc. and changed its name to Intellia Therapeutics, Inc. in July 2014. Intellia Therapeutics, Inc. was founded in 2014 and is headquartered in Cambridge, Massachusetts.
License and collaboration agreements with Novartis Institutes for BioMedical Research, Inc.; Regeneron Pharmaceuticals, Inc.; and Ospedale San Raffaele.
Precigen (PGEN)
Discovers and develops the next generation of gene and cellular therapies in the United States. It also provides disease-modifying therapeutics; genetically engineered swine for regenerative medicine applications; proprietary methane bioconversion platform that turns natural gas into energy and chemical products; and reproductive and embryo transfer technologies. In addition, the company offers UltraVector platform that incorporates advanced DNA construction technologies and computational models to design and assemble genetic components into complex gene expression programs; mbIL15, a gene that enhances functional characteristics of immune cells; Sleeping Beauty, a non-viral transposon/transposase system; AttSite recombinases, which breaks and rejoins DNA at specific sequences; AdenoVerse technology platform, a library of engineered adenovector serotypes; and L. lactis is a food-grade bacterium. Additionally, it provides RheoSwitch inducible gene switch that provides quantitative dose-proportionate regulation of the amount and timing of target protein expression; kill switches to selectively eliminate cell therapies in vivo; UltraCAR-T platform for the treatment of cancer; AdenoVerse Immunotherapy, a library of proprietary adenovectors for the gene delivery; and ActoBiotics platform, genetically modified bacteria that deliver proteins and peptides at mucosal sites.
Collaboration and license agreements with ZIOPHARM Oncology, Inc.; Ares Trading S.A.; Oragenics, Inc.; Intrexon T1D Partners, LLC; Intrexon Energy Partners, LLC; Intrexon Energy Partners II, LLC; Fibrocell Science, Inc.; OvaXon, LLC; S & I Ophthalmic, LLC; Harvest start-up entities; and Surterra Holdings, Inc. The company was formerly known as Intrexon Corporation and changed its name to Precigen, Inc. in January 2020. Precigen, Inc. was founded in 1998 and is based in Germantown, Maryland.
Pluristem (PSTI)
A bio-therapeutics company in Israel. It focuses on the research, development, clinical trial, and manufacture of cell therapeutic products and related technologies for the treatment of various ischemic, inflammatory, and hematologic conditions, as well as autoimmune disorders. The company develops PLacental eXpanded (PLX) cell therapy products, including PLX-PAD cells, which is Phase III clinical trial for the treatment of critical limb ischemia (CLI) in patients unsuitable for revascularization, recovery after surgery for hip fracture, and acute radiation syndrome (ARS), as well as peripheral and cardiovascular, and orthopedic diseases. It also develops PLX-R18 cells that is in Phase I clinical trial for incomplete hematopoietic recovery following hematopoietic cell transplantation, as well as conducts various animal studies for the evaluation of PLX-R18 for the treatment of ARS. The company has a license and commercialization agreement for conducting clinical trials and commercialization of its PLX-PAD product in South Korea related to the treatment CLI and intermediate claudication; and nTRACK, a collaborative project with Leitat to examine gold nano particles labeling of stem cells.
Collaboration agreements with the NASA's Ames Research Center to evaluate the potential of PLX cell therapies in preventing and treating medical conditions caused during space missions; and BIH Center for Regenerative Therapy and Berlin Center for Advanced Therapies to expand its framework and research agreement, as well as conduct a joint project evaluating the therapeutic effects of the registrant's patented PLX cell product candidates for treatment of the respiratory and inflammatory complications associated with the COVID-19 coronavirus. Pluristem Therapeutics Inc. was founded in 2001 and is based in Haifa, Israel.
uniQure NV (QURE)
uniQure NV, a gene therapy company, engages in the development and commercialization of treatments for patients suffering from genetic and other diseases in the Netherlands. Its lead program is Etranacogene dezaparvovec (AMT-061), which is in Phase III HOPE-B pivotal trial for the treatment of hemophilia B. The company also engages in developing AMT-130, a gene therapy that is in Phase I/II clinical study for the treatment of Huntington's disease; AMT-060, which is in Phase I/II clinical trial for the treatment of hemophilia B; AMT-180, an one-time intravenously-administered gene therapy candidate, which is in preclinical stage for the treatment of hemophilia A; and AMT-190, an IV-administered adeno-associated virus 5-based gene therapy for the treatment of Fabry disease. In addition, it engages in developing AMT-150, a gene therapy candidate for the treatment of Spinocerebellar Ataxia Type 3 disease.
Collaboration and license agreements with Bristol Myers-Squibb Company; Gen-X; and Synpromics Limited. uniQure N.V. was founded in 1998 and is headquartered in Amsterdam, the Netherlands.
Ultragenyx Pharmaceuticals (RARE)
Focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in the United States. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia; and Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII. The company is also developing small-molecule pipeline comprising UX007, a synthetic triglyceride for the treatment of long chain fatty-acid oxidation disorders, which is a set of rare metabolic diseases that prevents the conversion of fat into energy; and gene therapy pipeline consisting of DTX301, an adeno-associated virus 8 gene therapy product candidate for the treatment of patients with ornithine transcarbamylase, as well as DTX401, an AAV8 gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia. In addition, the company is developing UX068, which is in preclinical development for the treatment of creatine transporter deficiency (CTD); and UX053 for the treatment of glycogen storage disease type III.
Collaboration and license agreement with Arcturus Therapeutics Holdings Inc. to develop additional nucleic acid therapies for rare diseases. Ultragenyx Pharmaceutical Inc. was founded in 2010 and is headquartered in Novato, California.
ReNeuron (RENE.L)
Develops and sells cell-based therapies in the United Kingdom. The company engages in the development of CTX stem cell therapy candidate that is in Phase IIb clinical trial for stroke disability; and human retinal progenitor cell therapy that is in Phase I/IIa clinical trial for treating retinitis pigmentosa, a blindness-causing disease. It also developing CTX-derived exosomes, which are Nano-sized packages of information released by CTX cells. The company was founded in 1997 and is headquartered in Pencoed, the United Kingdom.
Regenxbio (RGNX)
Provides gene therapy product candidates to deliver genes to cells to address genetic defects or to enable cells in the body to produce therapeutic proteins or antibodies that are intended to impact disease. Its gene therapy product candidates are based on NAV Technology Platform, a proprietary adeno-associated virus gene delivery platform. The company's lead product candidate is RGX-314, which is in Phase I/IIa clinical trial for the treatment of wet age-related macular degeneration. It is also developing RGX-121 that is in Phase I/II clinical trial to treat mucopolysaccharidosis type II; RGX-111, which is in Phase I clinical trial for treating mucopolysaccharidosis type I; RGX-181 for the treatment of late infantile neuronal ceroid lipofuscinosis type II disease; and RGX-501, which is in Phase I/II clinical trials to treat homozygous familial hypercholesterolemia. The company was formerly known as ReGenX Biosciences, LLC and changed its name to REGENXBIO Inc. in September 2014. REGENXBIO Inc. was founded in 2008 and is headquartered in Rockville, Maryland.
REGENXBIO also licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies; and has a collaboration and license agreement with Neurimmune AG to develop vectorized antibodies for the treatment of neurodegenerative diseases
Sage Therapeutics (SAGE)
Develops and commercializes novel medicines to treat central nervous system (CNS) disorders. Its lead product candidate is ZULRESSO, a proprietary intravenous formulation of brexanolone that has completed Phase III clinical trials for the treatment of postpartum depression (PPD). The company's product pipeline also includes SAGE-217, a novel neuroactive steroid, which is in Phase III clinical trials for treating PPD, major depressive disorders, bipolar depression, and sleep disorders; and SAGE-324, a novel neuroactive steroid that has completed Phase I clinical trials to treat various neurological conditions, including essential tremor and epileptiform disorders. In addition, its product pipeline comprises SAGE-718, an oxysterol-based positive allosteric modulator of the NMDA receptor, which has completed Phase I clinical trial for the treatment of depression, Huntington's disease, Alzheimer's disease, attention deficit hyperactivity disorder, schizophrenia, and neuropathic pain; and SAGE-689, a novel GABAA receptor positive allosteric modulator that is in preclinical stage to treat acute and chronic CNS disorders. The company was formerly known as Sterogen Biopharma, Inc. and changed its name to Sage Therapeutics, Inc. in September 2011. Sage Therapeutics, Inc. was founded in 2010 and is headquartered in Cambridge, Massachusetts.
Strategic collaboration with Shionogi & Co., Ltd. for the development and commercialization of SAGE-217 in Japan, Taiwan, and South Korea.
Sangamo Therapeutics (SGMO)
Focuses on translating science into genomic medicines that transform patients' lives using platform technologies in gene therapy, cell therapy, genome editing, and genome regulation. The company's zinc finger proteins (ZFPs) could be engineered to make zinc finger nucleases (ZFNs), proteins that could be used to modify DNA sequences by adding or knocking out specific genes or genome editing; and ZFP transcription factors (ZFP TFs) proteins that can be used to increase or decrease gene expression. Its therapeutic products that are in Phase I/II clinical trial include SB-525 for the treatment of hemophilia A; ST-920 gene therapy for Fabry disease; ST-101 gene therapy for phenylketonuria; ST-400 and BIVV-003 for the treatment of hemoglobinopathies; SB-913 for the treatment of Mucopolysaccharidosis type II; SB-318 for the treatment of Mucopolysaccharidosis type I; and SB-FIX for the treatment of hemophilia B. The company was formerly known as Sangamo BioSciences, Inc. and changed its name to Sangamo Therapeutics, Inc. in January 2017. Sangamo Therapeutics, Inc. was founded in 1995 and is headquartered in Brisbane, California.
Collaborative and strategic partnerships with Biogen Inc.; Kite Pharma, Inc.; Pfizer Inc.; Sanofi Genzyme; Shire International GmbH; Dow AgroSciences LLC; Sigma-Aldrich Corporation; Genentech, Inc.; Open Monoclonal Technology, Inc.; F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc.; and California Institute for Regenerative Medicine.
Solid Biosciences (SLDB)
Engages in identifying and developing therapies for duchenne muscular dystrophy (DMD) in the United States. The company's lead product candidate is SGT-001, an adeno-associated viral vector-mediated gene transfer, which is in a Phase I/II clinical trial to restore functional dystrophin protein expression in patients' muscles. Its portfolio also comprises Anti-LTBP4, a monoclonal antibody that is in preclinical trials to reduce fibrosis and inflammation. In addition, the company engages in developing biomarkers and sensors; and Solid Suit program that includes the development of wearable assistive devices that focus on providing functional and therapeutic benefits to DMD patients. Solid Biosciences Inc. was founded in 2013 and is headquartered in Cambridge, Massachusetts.
Vericel (VCEL)
Researches, develops, manufactures, and distributes cellular therapies for sports medicine and severe burn care markets in the United States. It markets autologous cell therapy products comprising MACI, an autologous cellularized scaffold product for the repair of symptomatic, and single or multiple full-thickness cartilage defects of the knee; and Epicel, a permanent skin replacement humanitarian use device for the treatment of patients with deep-dermal or full-thickness burns. The company also develops ixmyelocel-T, a patient-specific multicellular therapy that has completed Phase IIb clinical trial for the treatment of advanced heart failure due to dilated cardiomyopathy. In addition, its preapproval stage product includes, NexoBrid, a registration-stage biological orphan product for debridement of severe thermal burns. The company was formerly known as Aastrom Biosciences, Inc. Vericel Corporation was founded in 1989 and is headquartered in Cambridge, Massachusetts.
Verastem Oncology (VSTM)
Focusing on developing and commercializing medicines to improve the survival and quality of life of cancer patients. It markets COPIKTRA (duvelisib), an oral inhibitor of phosphoinositide 3-kinase (PI3K) and dual inhibitor of PI3K-delta and PI3K-gamma, which is indicated for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma after at least two prior therapies and relapsed or refractory follicular lymphoma (FL) after at least two prior systemic therapies. The indication in FL is approved under accelerated approval based on overall response rate. In addition, it is developing the focal adhesion kinase inhibitor defactinib, which is being investigated in combination with immunotherapeutic agents for the treatment of various different cancer types, including pancreatic cancer, ovarian cancer, non-small cell lung cancer (NSCLC), mesothelioma, and other solid tumors.
Collaboration agreements with Yakult Honsha Co., Ltd. and CSPC Pharmaceutical Group Limited. The company was founded in 2010 and is headquartered in Needham, Massachusetts.
Voyager Therapeutics (VYGR)
http://www.voyagertherapeutics.com
A clinical-stage gene therapy company, focuses on the development of treatments for patients suffering from severe neurological diseases. The company's lead clinical candidate is the VY-AADC, which is in open-label Phase 1b clinical trial for the treatment of Parkinson's disease. Its preclinical programs comprise VY-SOD102 for the treatment of amyotrophic lateral sclerosis; VY-HTT01 for Huntington's disease; VY-FXN01 for Friedreich's ataxia; Tau program for the treatment of tauopathies, including Alzheimer's disease, progressive supranuclear palsy, and frontotemporal dementia; and alpha-synuclein program for synucleinopathies, Parkinson's disease, Lewy Body Dementia, and multiple system atrophy.
Strategic collaboration agreements with AbbVie Inc.; the University of Massachusetts; and ClearPoint Neuro, Inc., as well as collaborations with Brammer Bio and Fujifilm Diosynth Biotechnologies to support the development of its gene therapy programs. It also has a collaboration and license agreement with Neurocrine Biosciences, Inc. for the research, development, and commercialization of adeno-associated virus-based gene therapy products. Voyager Therapeutics, Inc. was founded in 2013 and is headquartered in Cambridge, Massachusetts.
