Taysha Gene Therapies, Inc. competitors & alternatives

Similar biotech company

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.

Atsena Therapeutics, Inc.BiotechnologyUnited States

70% matchPrivate

Biotechnology

Atsena Therapeutics, Inc. is a clinical-stage gene therapy company headquartered in Durham, North Carolina, established in 2019. The firm focuses on developing novel gene therapies to treat debilitating inherited blinding diseases using proprietary vector delivery technologies. Its scientific pipeline aims to address areas of high unmet medical need in ophthalmology.

Biotechnology

Gemma Labs Inc. (GEMMABio) is a biotechnology company that develops gene therapies for rare diseases using next-generation AAV technology. The company focuses on programs targeting motor neuron disorders, skeletal conditions, and cardiomyopathies to serve the rare disease community.

Similar biotech company

Passage Bio, Inc. is a biopharmaceutical company dedicated to pioneering genetic therapies for conditions affecting the central nervous system. Their advanced pipeline includes PBGM01, designed to treat infantile GM1 gangliosidosis by utilizing a proprietary AAVhu68 capsid to deliver a functional GLB1 gene, which codes for the lysosomal acid beta-galactosidase enzyme, to both the brain and peripheral tissues. Another key program, PBFT02, employs an AAV1 capsid to introduce a functional granulin (GRN) gene, encoding progranulin (PGRN), to the brain as a treatment for FTD-GRN. Additionally, PBKR03 is under development for infantile Krabbe disease, leveraging a proprietary AAVhu68 capsid to deliver a functional GALC gene, responsible for producing the hydrolytic enzyme galactosylceramidase, to the brain and surrounding tissues. The company's portfolio also features PBML04 for metachromatic leukodystrophy, PBAL05 for amyotrophic lateral sclerosis, and PBCM06 for Charcot-Marie-Tooth Type 2A. Passage Bio maintains a significant research collaboration with the Trustees of the University of Pennsylvania's Gene Therapy Program, alongside a development services and clinical supply agreement with Catalent Maryland, Inc. Established in 2017, the company's headquarters are located in Philadelphia, Pennsylvania.

Similar biotech company

CRISPR Therapeutics AG (CRSP) is a biotechnology firm dedicated to pioneering gene-based medicines for severe diseases. The company achieves this through its exclusive Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) technology, which enables precise and targeted alterations to an organism's genetic code. Its extensive pipeline includes therapeutic candidates spanning multiple medical areas, such as blood disorders (hemoglobinopathies), various cancers (oncology), regenerative medicine, and rare conditions. The company's flagship investigational therapy is CTX001, an ex vivo CRISPR gene-edited treatment. This therapy aims to benefit patients with transfusion-dependent beta-thalassemia or severe sickle cell disease by modifying their own hematopoietic stem cells to markedly boost the production of fetal hemoglobin within red blood cells. CRISPR Therapeutics is also advancing several other genetically engineered allogeneic (donor-sourced) CAR-T investigational therapies: CTX110, designed to combat cluster of differentiation 19-positive malignancies; CTX120, which targets B-cell maturation antigen for multiple myeloma that has relapsed or proven resistant to previous treatments; and CTX130, focused on Cluster of Differentiation 70 for a spectrum of solid tumors and blood cancers. Furthermore, the company is developing VCTX210, an immune-evasive, gene-edited stem cell-derived product candidate for treating type 1 diabetes. It is also pursuing various in vivo gene-editing initiatives aimed at addressing disorders affecting the liver, lungs, muscles, and central nervous system. The company has forged strategic alliances with significant partners, including Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, ViaCyte, Inc., Nkarta, Inc., and Capsida Biotherapeutics. CRISPR Therapeutics AG was established in 2013 and maintains its headquarters in Zug, Switzerland.

Coave TherapeuticsBiotechnologyFrance

69% match

Biotechnology

Coave Therapeutics is a clinical-stage biotechnology company headquartered in Paris, France. The company focuses on developing life-changing gene therapies targeting rare ocular and central nervous system diseases.

Biotechnology

Lexeo Therapeutics, Inc. is a genetic medicine firm currently in the clinical development phase, dedicated to addressing both inherited and acquired medical conditions. Its robust pipeline features several gene therapy candidates. These include LX2006, an AAVrh10-based therapy aimed at treating cardiomyopathy linked to Friedreich's ataxia (FA); LX2020, another AAVrh10-based candidate targeting arrhythmogenic cardiomyopathy; LX2021, designed for DSP cardiomyopathy; and LX2022, which focuses on hypertrophic cardiomyopathy (HCM) stemming from TNNI3 mutations. Furthermore, Lexeo is advancing LX1001, an AAVrh10-based gene therapy, alongside LX1020 and LX1021, all intended for individuals homozygous for APOE4. Additionally, LX1004 is under development to treat CLN2 Batten disease. Established in 2017, the company's headquarters are located in New York, New York.

Similar biotech company

Voyager Therapeutics, Inc. operates as a gene therapy company, concentrating its efforts on developing innovative treatments and pioneering advanced platform technologies. Its leading clinical asset, VY-AADC, is presently undergoing an open-label Phase 1 clinical trial for the management of Parkinson's disease. The company's preclinical portfolio is extensive, featuring VY-SOD102 for amyotrophic lateral sclerosis (ALS), VY-HTT01 for Huntington's disease, and VY-FXN01 for Friedreich's ataxia. Additionally, Voyager is pursuing a Tau program aimed at various tauopathies, including Alzheimer's disease, progressive supranuclear palsy, and frontotemporal dementia, alongside initiatives for spinal muscular atrophy. To advance its gene therapy product pipeline, the company has forged strategic collaboration and licensing agreements with key industry players such as Neurocrine Biosciences, Inc., Pfizer Inc., and Novartis Pharma, A.G. These partnerships cover the research, development, and commercialization of adeno-associated virus-based gene therapy products. Voyager Therapeutics, Inc. was founded in 2013 and maintains its corporate headquarters in Cambridge, Massachusetts.

Biotechnology

Beam Therapeutics Inc., founded in 2017 and based in Cambridge, Massachusetts, operates as a pioneering biopharmaceutical firm. Its core mission involves engineering precise genetic remedies to tackle a spectrum of severe human ailments, primarily within the United States. The company's developmental portfolio features several key candidates: BEAM-101 is being advanced to treat both sickle cell disease and beta thalassemia. BEAM-102 is specifically designed for addressing sickle cell disease. BEAM-201, an allogeneic chimeric antigen receptor T-cell therapy, is under investigation for individuals suffering from relapsed or refractory T-cell acute lymphoblastic leukemia. BEAM-301 is a liver-targeted candidate aimed at patients afflicted with Glycogen Storage Disease Type Ia. Beyond these flagship programs, Beam Therapeutics is also engaged in devising treatments for alpha-1 antitrypsin deficiency, various ocular conditions, and other disorders impacting the liver, muscular system, and central nervous system. To further its research and development objectives, Beam Therapeutics has forged numerous strategic alliances and partnerships. These include collaborations with Boston Children's Hospital; a research and clinical trial agreement with Magenta Therapeutics, Inc.; a licensing deal with Sana Biotechnology, Inc.; and a research partnership with the Institute of Molecular and Clinical Ophthalmology Basel. Additionally, the company maintains research collaborations with both Pfizer Inc. and Apellis Pharmaceuticals, Inc., alongside a comprehensive collaboration and license agreement with Verve Therapeutics, Inc.

Similar biotech company

GenSight Biologics is a clinical-stage biopharmaceutical company that develops gene therapies to treat neurodegenerative retinal diseases and central nervous system disorders. The company focuses on addressing conditions such as Leber Hereditary Optic Neuropathy (LHON), Retinitis Pigmentosa, and Geographic Atrophy using technologies like gene therapy, mitochondrial targeting sequences, and optogenetics.

Biotechnology

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.

AskBioBiotechnologyUnited States

67% match

Biotechnology

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.