Larimar Therapeutics, Inc.

Falcon BioBiotechnologyUnited States

73% match

Falcon Bio is a drug discovery company headquartered in Boston, Massachusetts, focused on targeting the root cause of cellular dysfunction to treat Friedreich's ataxia patients.

Design Therapeutics, Inc. is a biopharmaceutical company in the preclinical phase that focuses on creating treatments for genetic conditions stemming from nucleotide repeat expansions. Its current pipeline targets Friedreich's Ataxia (FA), a severe, progressive, monogenic, and autosomal recessive disease. FA primarily impairs mitochondrial function across various organ systems, resulting in neurological, cardiac, and metabolic complications. Another significant area of development is Myotonic Dystrophy Type-1 (DM1). This is a progressive, dominantly inherited monogenic neuromuscular illness that impacts skeletal muscle, the heart, brain, and other bodily systems. Beyond these, the company is advancing its GeneTAC product candidate platform to address an array of other monogenic conditions caused by nucleotide repeat expansions. These include Fragile X syndrome, various spinocerebellar ataxias, amyotrophic lateral sclerosis (ALS), frontotemporal dementia, Huntington's disease, and spinobulbar muscular atrophy. Established in 2017, Design Therapeutics, Inc. maintains its headquarters in Carlsbad, California.

Libra TherapeuticsLife SciencesUnited States

69% match

Libra Therapeutics is a biopharmaceutical company headquartered in Del Mar, California, focused on developing novel disease-modifying therapeutics. The company aims to restore cellular balance and improve lysosomal function to halt neurodegeneration in conditions such as amyotrophic lateral sclerosis.

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.

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.

ADARx Pharmaceuticals, Inc.ADRX · BiotechnologyUnited States

68% matchPublic

ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company located in San Diego, California. The company is committed to developing cutting-edge RNA-targeted therapeutics to treat various genetic and severe diseases. ADARx focuses on translating scientific research into life-saving medicines through its proprietary drug discovery pipeline.