Passage Bio, Inc. competitors & alternatives

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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.

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Praxis Precision Medicines, Inc. is a biopharmaceutical firm operating at the clinical stage, dedicated to creating innovative treatments for central nervous system (CNS) conditions that stem from an imbalance in neuronal activity. The company's primary investigational compounds currently undergoing evaluation include: PRAX-114, an extrasynaptic-preferring positive allosteric modulator of the GABAA receptor, which is currently in Phase IIa clinical trials for addressing both major depressive disorder and perimenopausal depression. Additionally, PRAX-944, a small molecule designed to selectively inhibit T-type calcium channels, is also progressing through Phase IIa clinical trials to manage essential tremor. Beyond these, Praxis is also advancing a pipeline of other promising drug candidates, such as: PRAX-562, a persistent sodium current blocker, undergoing Phase I clinical evaluation for treating severe pediatric epilepsy and adult cephalgia (headaches). PRAX-222, an antisense oligonucleotide (ASO) aimed at individuals suffering from gain-of-function (GOF) SCN2A epilepsy. And a program targeting KCNT1 for the treatment of KCNT1 GOF epilepsy. To further its research and development efforts, Praxis maintains several strategic alliances. These include a cooperation and licensing arrangement with RogCon Inc.; a licensing deal with Purdue Neuroscience Company; a comprehensive research collaboration, option, and license agreement with Ionis Pharmaceuticals, Inc.; and a collaborative project with The Florey Institute focused on the creation of three new antisense oligonucleotides (ASOs). Established in 2015, the company's headquarters are situated in Boston, Massachusetts.

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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.

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.

Taysha Gene Therapies, Inc.TSHA · BiotechDallas, United States

69% matchPublic

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Taysha Gene Therapies, Inc. is a biotech firm specializing in the creation and market introduction of gene therapies that utilize adeno-associated virus (AAV) vectors. Its core mission is to tackle inherited diseases affecting the central nervous system (CNS). The company's development pipeline features several key programs: TSHA-120 is aimed at giant axonal neuropathy; TSHA-102 is in development for Rett syndrome; TSHA-121 and TSHA-118 are both being advanced for CLN1 disease; TSHA-105 addresses SLC13A5 Deficiency; and TSHA-101 targets GM2 gangliosidosis. Furthermore, Taysha has forged a strategic alliance with The University of Texas Southwestern Medical Center to jointly advance and bring to market innovative gene therapy solutions. Founded in 2019, the company operates from its headquarters in Dallas, Texas.

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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.

Biotechnology

Precision BioSciences, Inc. is a clinical-stage biotechnology firm based in the United States, specializing in the creation of both in vivo gene editing solutions and ex vivo allogeneic CAR T-cell therapies. At its core is ARCUS, a proprietary genome editing platform designed to address and potentially cure genetic diseases. The company's portfolio also encompasses Ex vivo Allogeneic CAR T Immunotherapy, an innovative approach where specific immune cells, known as T-cells, are genetically modified outside the body to precisely identify and eliminate cancer cells. Among its prominent therapeutic candidates are: PBCAR0191, currently undergoing Phase 1/2a clinical trials for adult patients battling relapsed/refractory (R/R) non-Hodgkin lymphoma or R/R B-cell precursor acute lymphoblastic leukemia (B-ALL). PBCAR19B, an anti-CD19 CAR T candidate leveraging a "stealth cell" platform through a single-step gene edit, engineered to reduce the likelihood of chromosomal abnormalities. PBCAR269A, an investigational allogeneic CAR T immunotherapy formulated to target BCMA, intended for the treatment of R/R multiple myeloma. Precision BioSciences has strategically formed several collaborative alliances. These include an agreement with Les Laboratoires Servier for the development and commercialization of allogeneic chimeric antigen receptor T-cell therapies, aimed at various antigen targets, including hematological cancers beyond CD19, and solid tumors. The company is also partnering with Tiziana Life Sciences to assess foralumab, a fully human anti-CD3 monoclonal antibody, for its utility as a lymphodepleting agent in potential cancer treatments. Furthermore, a collaboration with iECURE, Inc. focuses on advancing ARCUS-based gene editing therapies. Established in 2006, Precision BioSciences, Inc. maintains its corporate headquarters in Durham, North Carolina.

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MeiraGTx Holdings plc operates as a clinical-stage gene therapy company, dedicated to pioneering therapeutic solutions for individuals afflicted by serious illnesses. Its development efforts span a broad spectrum of conditions, encompassing ocular diseases like inherited forms of blindness, as well as xerostomia (dry mouth) that often follows radiation treatment for head and neck cancers. The company also targets various degenerative and neurodegenerative disorders, notably amyotrophic lateral sclerosis (ALS) and Parkinson's disease. Currently, several of its programs are in active clinical development, including Phase 1/2 trials for Achromatopsia, X-Linked Retinitis Pigmentosa, RPE65-deficiency, radiation-induced Xerostomia, and Parkinson's. MeiraGTx is also preparing to commence a clinical program for xerostomia linked to Sjogren's syndrome and maintains preclinical initiatives for other neurodegenerative conditions. A key collaboration exists with Janssen Pharmaceuticals, Inc. to advance regulatable gene therapy treatments, leveraging MeiraGTx's proprietary riboswitch technology. Established in 2015, MeiraGTx Holdings plc is headquartered in New York, New York.

Code Biotherapeutics, Inc.BiotechnologyUnited States

68% matchPrivate

Biotechnology

Code Biotherapeutics, Inc. is a biotechnology company headquartered in the Greater Philadelphia area. The firm focuses on next-generation gene therapies and advanced medicine.

Prevail Therapeutics Inc.BiotechnologyUnited States

68% matchPrivate

Biotechnology

Prevail Therapeutics Inc. is a biotechnology company headquartered in New York City, New York, focused on developing gene therapies for patients with neurodegenerative diseases.

Ocugen, Inc.OCGN · BiotechMalvern, United States

68% matchPublic

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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.

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Krystal Biotech, Inc. is a clinical-stage biotechnology company focused on developing redosable gene therapies to address severe orphan diseases across the United States. Its most advanced therapeutic candidate, beremagene geperpavec (B-VEC), is currently undergoing Phase III clinical evaluation for the treatment of dystrophic epidermolysis bullosa. The company's pipeline also includes several other promising programs: KB105 is in Phase I/II clinical trials for patients suffering from deficient autosomal recessive congenital ichthyosis; KB301 is also in Phase I/II development, aiming to ameliorate wrinkles and other signs of aging or damaged skin. Additionally, Krystal Biotech is conducting preclinical research on KB407 for cystic fibrosis and KB104 for Netherton syndrome. In its earlier discovery phase, the company is exploring candidates such as KB5xx for chronic skin conditions and multiple KB3xx programs targeting aesthetic dermatological concerns. Krystal Biotech, Inc. was established in 2015 and is based in Pittsburgh, Pennsylvania.