Rezolute, Inc.

Crinetics Pharmaceuticals, Inc. operates as a clinical-stage pharmaceutical enterprise, dedicated to the identification, advancement, and market introduction of therapeutic solutions for infrequent endocrine conditions and related tumors. Their flagship drug candidate, Paltusotine, is an orally administered, selective, non-peptide somatostatin receptor type 2 agonist. This compound has concluded its Phase III clinical trials for the treatment of acromegaly, and has also completed Phase II trials targeting carcinoid syndrome and nonfunctional neuroendocrine tumors (NETs). Furthermore, the company's pipeline includes CRN04777, an oral selective non-peptide somatostatin type 5 receptor agonist, which is currently undergoing Phase I clinical trials for congenital hyperinsulinism. Another investigational drug, CRN04894, an oral adrenocorticotrophic hormone antagonist, is in Phase I clinical trials for Cushing's disease and congenital adrenal hyperplasia. Crinetics Pharmaceuticals, Inc. was founded in 2008 and maintains its headquarters in San Diego, California.

Endsulin, Inc. is a biotechnology company developing a liver-directed gene therapy for Type 1 Diabetes. Its proprietary technology aims to re-engineer liver cells to restore the body's natural ability to produce and regulate insulin, eliminating the need for artificial insulin delivery methods.

Travere Therapeutics, Inc.TVTX · BiotechnologySan Diego, United States

65% matchPublicAI 6/100 ▼Green 0/100

Travere Therapeutics is a biopharmaceutical company headquartered in San Diego, California, focused on identifying, developing, and delivering life-changing therapies for people living with rare diseases.

Rhythm Pharmaceuticals, Inc.RYTM · BiotechBoston, United States

64% matchPublic

Rhythm Pharmaceuticals, Inc. is a biopharmaceutical company with commercial products, dedicated to the discovery, development, and market launch of therapies addressing rare genetic conditions that cause obesity. Its leading pharmaceutical, IMCIVREE, functions as a powerful agonist of the melanocortin-4 receptor (MC4R). This medication is indicated for treating obesity stemming from deficiencies in pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or the leptin receptor (LEPR), alongside its use in patients with Bardet-Biedl and Alström syndromes. Furthermore, the company is progressing setmelanotide (the active compound in IMCIVREE) through Phase II clinical trials. These studies are evaluating its potential for a broader spectrum of applications, including obesity caused by heterozygous POMC or LEPR deficiencies, steroid receptor coactivator 1 (SRC1) deficiency, SH2B1 deficiency, MC4 receptor deficiency, and obesity associated with Smith-Magenis syndrome, POMC epigenetic disorders, and other MC4R-related conditions. Rhythm Pharmaceuticals holds a collaborative research agreement with the Clinical Registry Investigating Bardet-Biedl Syndrome. The firm, headquartered in Boston, Massachusetts, was established in 2008 and changed its name from Rhythm Metabolic, Inc. to Rhythm Pharmaceuticals, Inc. in October 2015.

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.

OrsoBio, Inc.BiotechnologyUnited States

64% match

OrsoBio, Inc. is a privately held, clinical-stage biopharmaceutical company dedicated to developing therapies to treat severe metabolic disorders.