California Institute of Regenerative Medicine

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cellular therapies to treat unmet medical needs. The company is focused on developing new therapeutics for age-related degenerative disease. BioTime and its subsidiaries are developing OpRegen® (a cell-based therapy for age-related macular degeneration), therapies for cancer, and arthritis, respectively. Our subsidiary ReCyte Therapeutics is using proprietary technology to reverse the developmental aging of human cells to manufacture young vascular progenitors for the treatment of age-related vascular disease. Relatively near-term product development by BioTime and its subsidiaries include Renevia, a patented hyaluronan-based hydrogel device for cell delivery; PanC-Dx, a blood-based pan-cancer screening diagnostic; and re-starting the embryonic stem cell-based clinical trial for spinal cord injury that was originally begun by Geron, using the stem cell assets acquired from them in 2013. BioTime also markets an FDA-approved blood plasma expander called Hextend®, which is currently marketed in collaboration with Hospira.

Rubedo Life Sciences, Inc. is a biopharmaceutical company based in Sunnyvale, California, focused on developing therapies for age-related diseases by targeting senescent cells. Incorporated in 2018, the company utilizes its ALEMBIC drug discovery platform to create a pipeline of therapeutic candidates that selectively eliminate senescent cells, as well as pro-inflammatory and pro-fibrotic cell populations resulting from cellular stress and damage. By promoting selective apoptosis in aged or pathological tissues, Rubedo aims to regenerate tissues, improve the function of healthy cells, and ultimately extend the health span of patients. The company is dedicated to addressing the challenges posed by age-related conditions through innovative therapeutic solutions.

Immusoft Corporation is a biotechnology company based in Seattle, Washington, focused on developing autologous cell therapies for various human diseases through its proprietary Immune System Programming (ISP) technology. Founded in 2009, the company modifies a patient's immune cells, specifically B cells, to create miniature drug factories that produce gene-encoded medicines, or biologics. By reprogramming these cells, Immusoft's technology enables the in vivo delivery of therapeutics that can replace missing or defective enzymes and proteins. The core ISP technology was initially developed at the California Institute of Technology and is exclusively licensed to Immusoft. This innovative approach aims to improve treatments for rare and infectious diseases by harnessing the body's own immune system.

Juvena Therapeutics, Inc., a biopharmaceutical company, develops protein-based tissue rejuvenation therapeutics for elderly people. Its products are used for treatment of sarcopenia, wound healing, and neural regeneration. Juvena Therapeutics, Inc. was founded in 2017 and is headquartered in San Mateo, California.

Poseida Therapeutics, Inc. is a clinical-stage biopharmaceutical company headquartered in San Diego, California, focused on developing innovative therapies for patients with significant unmet medical needs. The company is advancing a diverse pipeline that includes treatments for hematological malignancies and solid tumors, as well as liver-directed gene therapies aimed at rare diseases. Poseida is known for its proprietary gene engineering platforms, including the non-viral piggyBac DNA Modification System and the Cas-CLOVER site-specific gene editing system, which facilitate the creation of next-generation cell and gene therapeutics. Additionally, the company's portfolio encompasses CAR-T therapies for cancer and gene therapies targeting rare and life-threatening conditions, such as Ornithine transcarbamylase deficiency and methylmalonic acidemia. Founded in 2014, Poseida Therapeutics is committed to addressing critical health challenges through its advanced therapeutic solutions.

Sangamo Therapeutics is a clinical-stage biotechnology company focused on developing genomic therapies aimed at improving the lives of patients with serious health conditions. The company utilizes advanced technologies in genome editing, gene therapy, gene regulation, and cell therapy to create innovative treatments. Sangamo's product pipeline addresses various medical areas, including hemophilia, central nervous system disorders, HIV, lysosomal storage disorders, and hemoglobinopathies. Additionally, the company has a priority neurology pipeline that includes therapies for chronic neuropathic pain, prion disease, and other neurological conditions, along with partnered programs targeting hemophilia A, oncology, amyotrophic lateral sclerosis, and Huntington's disease. Sangamo aims to leverage its scientific expertise to deliver transformative medicines for patients and their families.

Fate Therapeutics is using the fundamental biological mechanisms that guide cell fate to develop stem cell therapeutics. The company has brought together the foremost scientists from the nation's research hotbeds (Boston, San Francisco, San Diego, and Seattle) who have demonstrated the potential to create and modulate stem cells to restore health. The backstory is that while others were working towards therapies based on transplanting stem cell-derived cells into patients, Randall Moon (HHMI and the University of Washington) and Alex Rives (Arch Venture Capitol) envisioned a different approach to develop regenerative therapies. The concept, based on data from hematopoietic progenitor cells, was that one could modulate the properties of stem cells in vivo using small molecules or biologics. They presented this idea to Arch Venture Capitol in Seattle, which launched the company, soon to be joined by other investors.

Medeor Therapeutics, Inc. specializes in the research, development, and commercialization of personalized cellular immunotherapies aimed at enhancing outcomes for organ transplant recipients. The company focuses on creating innovative cellular immunotherapy products, particularly for kidney transplant patients, under the MDR-10X brand. By employing advanced approaches to organ transplant immune tolerance and immuno-oncology, Medeor Therapeutics seeks to improve kidney function and reduce the risk of graft rejection more effectively than traditional immunosuppressive medications. Founded in 2012, the company is headquartered in San Mateo, California.

ViaCyte, Inc. is a regenerative medicine company based in San Diego, California, focused on developing innovative cell replacement therapies for diabetes. The company is working on several product candidates, including VC-01, designed for type 1 diabetes, and VC-02, which facilitates blood vessel interaction with implanted pancreatic precursor cells. Their VCTX210 product utilizes a proprietary human stem cell line that helps prevent immune system rejection, eliminating the need for immunosuppressants. ViaCyte's therapies differentiate stem cells into pancreatic beta-cell precursors that are implanted in an encapsulation device, enabling the production of insulin in response to blood glucose levels. The company's aim is to provide long-term solutions for both type 1 and type 2 diabetes patients, reducing their dependence on insulin and minimizing associated health complications. Founded in 1999 and formerly known as Novocell, Inc., ViaCyte also has operations in Athens, Georgia.

Poseida Therapeutics, Inc. is a clinical-stage biopharmaceutical company headquartered in San Diego, California, focused on developing innovative therapies for patients with significant unmet medical needs. The company is advancing a diverse pipeline that includes treatments for hematological malignancies and solid tumors, as well as liver-directed gene therapies aimed at rare diseases. Poseida is known for its proprietary gene engineering platforms, including the non-viral piggyBac DNA Modification System and the Cas-CLOVER site-specific gene editing system, which facilitate the creation of next-generation cell and gene therapeutics. Additionally, the company's portfolio encompasses CAR-T therapies for cancer and gene therapies targeting rare and life-threatening conditions, such as Ornithine transcarbamylase deficiency and methylmalonic acidemia. Founded in 2014, Poseida Therapeutics is committed to addressing critical health challenges through its advanced therapeutic solutions.

Humacyte, Inc. is a medical research and development company based in Morrisville, North Carolina, focused on creating and commercializing human tissue-based products for regenerative medicine and vascular surgery. Founded in 2004, the company specializes in developing acellular extracellular matrices derived from banked vascular smooth muscle cells, which are decellularized to prevent rejection. These matrices serve as tissue-engineered grafts, providing solutions for patients requiring vascular repair or replacement. Humacyte's innovative biotechnology platform aims to deliver universally implantable bioengineered human tissues and organs, addressing a variety of diseases, injuries, and chronic conditions, ultimately seeking to enhance patient outcomes and transform medical practices.

Brainstorm Cell Therapeutics Inc., a biotechnology company, 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 has a partnership with Catalent for the manufacture of NurOwn, an autologous cellular therapy. The company was formerly known as Golden Hand Resources Inc. and changed its name to Brainstorm Cell Therapeutics Inc. in November 2004. Brainstorm Cell Therapeutics Inc. was founded in 2000 and is headquartered in New York, New York.

Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. Operating in the United Kingdom, European Union, and the United States, Orchard employs an autologous ex vivo gene therapy approach to transform patients’ hematopoietic stem cells into gene-modified products for treatment through a single administration. Its portfolio includes Strimvelis, the first approved gene therapy for adenosine deaminase-severe combined immunodeficiency (ADA-SCID), along with several products in clinical development targeting metachromatic leukodystrophy, Wiskott-Aldrich syndrome, X-linked chronic granulomatous disease, and transfusion-dependent beta-thalassemia. The company also has a robust preclinical pipeline addressing various mucopolysaccharidosis types. Orchard collaborates with leading institutions in gene therapy, enhancing its research capabilities and clinical programs. Founded in 2015, Orchard Therapeutics continues to demonstrate a commitment to transforming the lives of patients through its advanced therapies.

Semma Therapeutics, Inc. is focused on developing innovative cell therapies for patients with Type 1 diabetes, who traditionally rely on insulin injections. Founded based on groundbreaking research by Professor Douglas Melton, the company has licensed a method to generate functional, insulin-producing beta cells in the laboratory. Semma Therapeutics aims to combine these proprietary cells with advanced devices to create a comprehensive therapy that can replace the missing beta cells without the need for immunosuppression. By striving to bring this transformative treatment to clinical application, Semma Therapeutics seeks to significantly enhance the lives of diabetes patients. Headquartered in Cambridge, Massachusetts, the company is a subsidiary of Vertex Pharmaceuticals and collaborates strategically with Defymed.

Humacyte, Inc. is a medical research and development company based in Morrisville, North Carolina, focused on creating and commercializing human tissue-based products for regenerative medicine and vascular surgery. Founded in 2004, the company specializes in developing acellular extracellular matrices derived from banked vascular smooth muscle cells, which are decellularized to prevent rejection. These matrices serve as tissue-engineered grafts, providing solutions for patients requiring vascular repair or replacement. Humacyte's innovative biotechnology platform aims to deliver universally implantable bioengineered human tissues and organs, addressing a variety of diseases, injuries, and chronic conditions, ultimately seeking to enhance patient outcomes and transform medical practices.

Capricor Therapeutics is a clinical-stage biotechnology company headquartered in Beverly Hills, California, specializing in the discovery, development, and commercialization of biological therapies for the treatment of diseases, particularly Duchenne muscular dystrophy (DMD) and other rare disorders. The company's lead product candidate, CAP-1002, is an allogeneic cell therapy currently in phase II clinical trials aimed at treating late-stage DMD. Additionally, Capricor has explored CAP-1002 for other cardiac conditions, including heart failure and post-myocardial infarction complications. The company is also developing CAP-2003, which is in pre-clinical development for inflammatory conditions, alongside other innovative therapeutic approaches, including exosome-based treatments and vaccines. Founded in 2005, Capricor Therapeutics is committed to addressing unmet medical needs through its advanced therapeutic programs.

ImmunoCellular Therapeuti is a clinical-stage biotechnology company. It is engaged in developing immune-based therapies for the treatment of cancers which can elicit an immune response against several antigens. The company has developed ICT-107, which is a dendritic cell (DC) immunotherapy for the treatment of newly diagnosed glioblastoma multiforme (GBM), which is a lethal type of brain cancer. It develops Immunotherapies for treating cancer in which a patient's own immune system is stimulated to target tumor antigens, which are molecular signals that the immune system uses to identify foreign bodies.

ViaCyte, Inc. is a regenerative medicine company based in San Diego, California, focused on developing innovative cell replacement therapies for diabetes. The company is working on several product candidates, including VC-01, designed for type 1 diabetes, and VC-02, which facilitates blood vessel interaction with implanted pancreatic precursor cells. Their VCTX210 product utilizes a proprietary human stem cell line that helps prevent immune system rejection, eliminating the need for immunosuppressants. ViaCyte's therapies differentiate stem cells into pancreatic beta-cell precursors that are implanted in an encapsulation device, enabling the production of insulin in response to blood glucose levels. The company's aim is to provide long-term solutions for both type 1 and type 2 diabetes patients, reducing their dependence on insulin and minimizing associated health complications. Founded in 1999 and formerly known as Novocell, Inc., ViaCyte also has operations in Athens, Georgia.

Bluebird Bio is a clinical-stage biotechnology company focused on developing transformative gene therapies for severe genetic diseases and cancer. The company is engaged in researching and commercializing therapies that aim to genetically modify patients' cells to correct the underlying genetic causes of diseases. Key programs include LentiGlobin for β-thalassemia and sickle cell disease, and Lenti-D for cerebral adrenoleukodystrophy. In oncology, bluebird bio is developing CAR T cell therapies, such as bb2121 and bb21217, for multiple myeloma. The company has formed strategic collaborations with Bristol-Myers Squibb and Regeneron Pharmaceuticals to advance gene therapies in oncology, and works with various partners to develop innovative solutions in cancer treatment and in vivo genome editing for genetic disorders. Founded in 1992 and headquartered in Cambridge, Massachusetts, bluebird bio generates revenue through collaboration arrangements, research fees, license fees, and grants.

VistaGen Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative medicines for central nervous system disorders, particularly depressive and social anxiety disorders. The company’s primary pipeline includes PH94B, a neuroactive nasal spray currently preparing for Phase III clinical trials aimed at treating acute anxiety in adults with social anxiety disorder, and PH10, which is moving toward Phase 2b development for major depressive disorder. Additionally, AV-101, an orally available prodrug, is in Phase 2 development for various conditions including major depressive disorder, suicidal ideation, neuropathic pain, levodopa-induced dyskinesia, and epilepsy. VistaGen has established collaborations and licensing agreements with several organizations to enhance its research and development efforts, particularly for expanding the clinical applications of PH94B in Asia. Founded in 1998 and headquartered in South San Francisco, California, VistaGen is committed to addressing significant unmet needs in mental health care.