REGENXBIO Inc.

AskBioBiotechnologyUnited States

71% match

AskBio is a clinical-stage gene therapy company that develops adeno-associated virus (AAV) gene therapies to treat genetic and complex disorders. The company focuses on advancing genetic medicine through its proprietary technology platforms, manufacturing processes, and clinical trials.

Rocket Pharmaceuticals, Inc., operating alongside its affiliates, is an innovative biotechnology enterprise dedicated to pioneering gene therapies for rare and severely debilitating diseases. The company's active clinical pipeline features multiple promising programs. It is currently progressing three ex vivo lentiviral vector therapies aimed at specific genetic conditions: Fanconi anemia, characterized by a bone marrow defect that hinders blood cell production; leukocyte adhesion deficiency-I, an inherited disorder resulting in a compromised immune system; and pyruvate kinase deficiency, a rare autosomal recessive red blood cell ailment leading to chronic non-spherocytic hemolytic anemia. Complementing these, Rocket Pharmaceuticals also has an in vivo adeno-associated virus program underway for Danon disease, a serious multi-organ lysosomal storage disorder that tragically often results in early mortality due to heart failure. To facilitate its extensive research and development efforts, the company has established key licensing partnerships with esteemed institutions such as the Fred Hutchinson Cancer Research Center, European research bodies including CIEMAT and its collaborators (Centro de Investigacion Biomedica En Red and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz), UCL Business PLC (in conjunction with CIEMAT), The Regents of the University of California, and REGENXBIO, Inc. Rocket Pharmaceuticals, Inc. maintains its primary corporate location in Cranbury, New Jersey.

4D Molecular Therapeutics, Inc. is a clinical-stage gene therapy company that engineers therapeutic agents by leveraging its proprietary adeno-associated virus (AAV) vector technology. The company's developmental efforts are concentrated across a trio of therapeutic fields: ophthalmology (eye conditions), cardiology (heart ailments), and pulmonology (respiratory disorders). Within its active pipeline, three specific product candidates have advanced into clinical trial phases, all currently undergoing Phase 1/2 clinical assessment: 4D-125 is being evaluated for the treatment of X-linked retinitis pigmentosa. 4D-110 targets choroideremia. 4D-310 is focused on addressing Fabry disease. Beyond these, two Investigational New Drug (IND) candidates are in development: 4D-150, aimed at combating wet age-related macular degeneration, and 4D-710, designed to tackle cystic fibrosis lung disease. To advance its mission, 4D Molecular Therapeutics, Inc. actively engages in research and development partnerships with entities such as uniQure, CRF, Roche, and CFF. The firm commenced operations in 2013 and maintains its headquarters in Emeryville, California.

Intellia Therapeutics, Inc. is a biotechnology firm dedicated to advancing therapeutic treatments through its expertise in genome editing. The company's pipeline includes several in vivo (administered within the body) programs. NTLA-2001 is currently undergoing a Phase 1 clinical trial for transthyretin amyloidosis, while NTLA-2002 targets hereditary angioedema. Additionally, Intellia is developing various other liver-focused therapies for conditions such as hemophilia A and B, hyperoxaluria Type 1, and alpha-1 antitrypsin deficiency. Its ex vivo (processed outside the body) pipeline features NTLA-5001, a candidate for acute myeloid leukemia. The company is also progressing proprietary programs focused on creating engineered cell therapies to address diverse oncological and autoimmune disorders. At the core of Intellia's technology is the Clustered, Regularly Interspaced Short Palindromic Repeats/CRISPR associated 9 (CRISPR/Cas9) system. Intellia Therapeutics, Inc. has established multiple licensing and collaboration agreements. These include a partnership with Novartis Institutes for BioMedical Research, Inc. to engineer hematopoietic stem cells for treating sickle cell disease, and a co-development agreement with Regeneron Pharmaceuticals, Inc. for potential products against hemophilia A and B. The company also collaborates with Ospedale San Raffaele and holds a strategic alliance with SparingVision SAS to develop novel genomic medicines for ocular diseases, leveraging CRISPR/Cas9 technology. The company, initially known as AZRN, Inc., was founded in 2014 and operates from its headquarters in Cambridge, Massachusetts.

Ocugen, Inc.OCGN · BiotechMalvern, United States

68% matchPublic

Ocugen, Inc. is a biopharmaceutical company in the clinical development phase, primarily dedicated to pioneering gene therapies aimed at treating various ophthalmic conditions leading to blindness. The company's developmental pipeline features several key candidates: OCU400: An innovative gene therapy designed to reinstate both the function and structural integrity of the retina across a broad spectrum of genetically diverse inherited retinal diseases, such as retinitis pigmentosa and Leber congenital amaurosis. OCU410: Another gene therapy candidate, currently being developed to address dry age-related macular degeneration (AMD). OCU200: A novel fusion protein, presently undergoing preclinical evaluation for its potential in treating diabetic macular edema, diabetic retinopathy, and wet AMD. Ocugen maintains significant strategic collaborations, including a partnership with CanSino Biologics Inc. for the joint development and manufacturing of gene therapies, and an alliance with Bharat Biotech for the commercialization of COVAXIN within the United States market. The company's corporate headquarters are situated in Malvern, Pennsylvania.

Neurogene Inc. is dedicated to engineering transformative genetic treatments for individuals and their families grappling with debilitating neurological conditions. Its current pipeline prominently features two key investigational therapies: NGN-401, an AAV9 gene therapy specifically targeting Rett syndrome, and NGN-101, which is being developed to address neuronal ceroid lipofuscinosis subtype 5 (Batten disease). The company's operations are based in New York, New York.