Rhythm Pharmaceuticals, Inc.

Relay Therapeutics, Inc. is a precision medicine company currently engaged in clinical trials. Its central mission involves revolutionizing the drug discovery process, with a particular focus on enhancing the creation of small molecule therapies for specific types of cancer and genetic conditions. The company's pipeline features several promising drug candidates: RLY-4008, an orally administered small molecule designed to inhibit fibroblast growth factor receptor 2 (FGFR2), which is undergoing its initial human trials for patients with advanced or metastatic solid tumors exhibiting FGFR2 alterations; RLY-2608, a leading program targeting mutant phosphoinositide 3 kinase alpha (PI3Ka); and RLY-1971, another oral small molecule, an inhibitor of SHP2 (protein tyrosine phosphatase Src homology region 2 domain-containing phosphatase-2), currently in Phase 1 trials for individuals with advanced solid tumors. Relay Therapeutics has established strategic partnerships, including a collaboration with D. E. Shaw Research, LLC, which utilizes computational modeling to analyze protein motion for the identification and development of therapeutic compounds. Furthermore, an agreement with Genentech, Inc. supports the development and commercialization of RLY-1971. Founded in 2015 as Allostery, Inc., the company rebranded to Relay Therapeutics, Inc. in December of that year and maintains its headquarters in Cambridge, Massachusetts.

Beacon TherapeuticsBiotechnology

66% match

Beacon Therapeutics is a clinical-stage biotechnology company dedicated to saving and restoring sight for people living with rare and prevalent ocular diseases. The company works to deliver advanced retinal gene therapies to patients experiencing blindness.

Stoke Therapeutics, Inc. is an emerging biopharmaceutical company dedicated to developing innovative antisense oligonucleotide (ASO) therapies. Its primary focus is to target and address the fundamental causes of severe genetic disorders, particularly within the United States. The company employs its exclusive Targeted Augmentation of Nuclear Gene Output (TANGO) platform to engineer ASOs that precisely enhance protein expression. Stoke's most advanced candidate, STK-001, is currently progressing through Phase I/IIa clinical trials for Dravet syndrome, a severe and debilitating genetic epilepsy. Additionally, STK-002 is in the preclinical development stage for treating autosomal dominant optic atrophy. The company has also established a licensing and collaborative agreement with Acadia Pharmaceuticals Inc. to identify, develop, and market new RNA-based medicines for critical and rare genetic neurodevelopmental conditions impacting the central nervous system. Formerly known as ASOthera Pharmaceuticals, Inc., the company rebranded to Stoke Therapeutics, Inc. in May 2016. Founded in 2014, Stoke Therapeutics, Inc. maintains its headquarters in Bedford, Massachusetts.

Rinascera Therapeutics, Inc.BiotechnologyUnited States

65% match

Rinascera Therapeutics, Inc. is a clinical-stage biotechnology company located in Boston, Massachusetts, dedicated to transforming the treatment of rare genetic skin diseases.

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.

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.