Avalo Therapeutics, Inc.

Abeona Therapeutics Inc. operates as a clinical-stage biopharmaceutical company, specializing in the creation of gene and cell therapies to combat critical, uncommon genetic illnesses. Their foremost developmental asset is EB-101, a personalized gene-corrected cell therapy that has progressed to Phase III clinical trials for the treatment of recessive dystrophic epidermolysis bullosa. The company's pipeline also encompasses several other programs: ABO-102, an adeno-associated virus (AAV)-based gene therapy designed for Sanfilippo syndrome type A; ABO-201, aimed at CLN3 disease; ABO-401, targeting cystic fibrosis; and ABO-50X, intended for genetic eye disorders. Additionally, Abeona actively advances AAV-based gene therapies through its proprietary AIM vector platform. Incorporated in 1974, the firm was initially named PlasmaTech Biopharmaceuticals, Inc. before officially adopting the name Abeona Therapeutics Inc. in June 2015. The company's corporate base is situated in New York, New York.

REGENXBIO Inc. is a biotechnology company operating in the clinical stage, dedicated to developing gene therapy candidates. These therapies are designed to introduce genes into cells, aiming either to rectify genetic deficiencies or to stimulate the body's own cells to produce therapeutic proteins or antibodies to combat diseases. Central to its therapeutic development is the proprietary NAV Technology Platform, an adeno-associated virus-based gene delivery system. The company's primary investigational asset, RGX-314, is currently in Phase III clinical trials for treating wet age-related macular degeneration. Its pipeline also includes RGX-121 and RGX-111, both in Phase I/II trials for mucopolysaccharidosis type II and type I, respectively; RGX-181, which is in preclinical development for late-infantile neuronal ceroid lipofuscinosis type II disease; RGX-202, undergoing Phase I/II evaluation for Duchenne muscular dystrophy; and RGX-381, at the preclinical stage for addressing the ocular manifestations of CLN2 disease. In addition to its internal programs, REGENXBIO Inc. licenses its NAV Technology Platform to other biotech and pharmaceutical enterprises. It also maintains a collaborative and licensing agreement with Neurimmune AG for the advancement of innovative gene therapies. Founded in 2008, REGENXBIO Inc. maintains its headquarters in Rockville, Maryland.

Aditxt, Inc. is a biotechnology firm dedicated to advancing immune system health through innovative mapping and reprogramming techniques. The company's developments include AditxtScore, a platform designed to empower individuals by providing insights into their immune profiles for better management and informed awareness of immune system attacks or dysfunctions. Aditxt also pioneers Apoptotic DNA Immunotherapy, a nucleic acid-based solution that emulates the body's inherent mechanisms for developing self-tolerance to its own tissues. Additionally, Aditxt is actively researching and developing ADi products with diverse therapeutic applications, such as for organ transplant procedures (including skin grafting), various autoimmune conditions, and allergic reactions. The company holds licensing agreements with esteemed institutions like Loma Linda University and Leland Stanford Junior University. Established in 2017, this Richmond, Virginia-headquartered company was initially known as ADiTx Therapeutics, Inc. before rebranding to Aditxt, Inc. in July 2021.

A2 Biotherapeutics, Inc.BiotechnologyUnited States

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A2 Biotherapeutics, Inc. is a clinical-stage biotechnology research company headquartered in Agoura Hills, California. The company develops precision T-cell therapies designed to target solid tumors while sparing healthy tissue.

Allogene Therapeutics, Inc. operates as a clinical-stage immuno-oncology firm dedicated to the creation and commercialization of genetically engineered allogeneic T-cell therapies for the treatment of various cancers. A pivotal product in their development pipeline is UCART19, an allogeneic chimeric antigen receptor (CAR) T-cell therapy. This candidate is being developed, manufactured, and prepared for market release to address relapsed/refractory (R/R) CD19-positive B-cell acute lymphoblastic leukemia (ALL) in both children and adults. The company's portfolio also encompasses ALLO-501, an anti-CD19 allogeneic CAR T-cell candidate currently in Phase I clinical trials for R/R non-Hodgkin lymphoma. A related therapeutic, ALLO-501A, is progressing through Phase I/II studies, targeting R/R large B-cell lymphoma or transformed follicular lymphoma. Furthermore, Allogene is advancing several other promising candidates: ALLO-715, an allogeneic CAR T-cell therapy in Phase I for R/R multiple myeloma; ALLO-605, another allogeneic CAR T-cell designed for multiple myeloma; and ALLO-647, an anti-CD52 monoclonal antibody. Their investigational therapies also include those targeting CD70 for renal cell cancer, ALLO-819 (an allogeneic CAR T-cell treatment) for acute myeloid leukemia, and DLL3 for small cell lung cancer and other aggressive neuroendocrine tumors. Allogene maintains a robust network of strategic alliances, which includes licensing and collaboration agreements with organizations such as Pfizer Inc., Servier, Cellectis S.A., and Notch Therapeutics Inc. They also hold a clinical trial collaboration with SpringWorks Therapeutics, Inc. Additionally, a strategic partnership with The University of Texas MD Anderson Cancer Center supports the preclinical and clinical evaluation of their allogeneic CAR T-cell product pipeline. Founded in 2017, the company's corporate headquarters are located in South San Francisco, California.

Precigen, Inc. is an American company dedicated to the discovery and development of cutting-edge gene and cellular therapies. The company also offers disease-modifying treatments, genetically engineered swine for regenerative medicine, and advanced reproductive and embryo transfer technologies. Its extensive portfolio of proprietary platforms includes UltraVector, which leverages advanced DNA construction and computational modeling to engineer complex gene expression programs; mbIL15, a gene designed to enhance immune cell function; the non-viral Sleeping Beauty transposon/transposase system for genetic modification; and AttSite recombinases, tools for precise DNA recombination. Furthermore, Precigen utilizes its AdenoVerse platform, a library of engineered adenovectors for gene delivery and immunotherapy applications, alongside L. lactis, a food-grade bacterium. The company also provides RheoSwitch, an inducible gene switch system offering quantitative, dose-proportionate control over target protein expression's timing and amount; kill switches, designed to selectively eliminate cell therapies within the body; tissue-specific promoters for targeted gene expression; the UltraCAR-T platform for cancer treatment; and the ActoBiotics platform, which employs genetically modified bacteria to deliver proteins and peptides directly to mucosal sites. Precigen maintains strategic collaboration and license agreements with Alaunos Therapeutics, Inc., Ares Trading S.A., Oragenics, Inc., Castle Creek Biosciences, Inc., and Intrexon Energy Partners I and II, LLC. Established in 1998, Precigen, Inc., previously known as Intrexon Corporation, adopted its current name in January 2020 and is headquartered in Germantown, Maryland.