-- Leading mitochondrial myopathies companies commercializing and developing emerging therapies, including Stealth BioTherapeutics, UCB, Khondrion, PTC Therapeutics, Pharming Technologies, Tisento ...

DURHAM, N.C.--(BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), an advanced gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies ...

Trial Results Support Transition into Late-Stage Development with Program Expected to be Phase 2b/3-ready in H2 2026BERLIN, GERMANY, January 29, ...

- Data presented at Mitochondrial Medicine Therapeutic Development (MMTD) annual conference demonstrated ARCUS’ ability to efficiently eliminate mutant mitochondrial DNA without nuclear off-target ...

The oxidase, cytochrome c, assembly 1-like (OXA1L) gene plays a key role in inserting and assembling proteins in the inner mitochondrial membrane, and recent studies have linked some variants to ...

Researchers have discovered a mechanism in our cells that is essential to energy production in the muscles. The discovery may lead to new treatment for disorders affecting the muscles, such as ...

IRVINE, Calif., Dec. 14, 2023 (GLOBE NEWSWIRE) -- Reneo Pharmaceuticals, Inc. (NASDAQ: RPHM), a pharmaceutical company focused on the development and commercialization of therapies for patients with ...

IRVINE, Calif., March 14, 2023 (GLOBE NEWSWIRE) -- Reneo Pharmaceuticals, Inc. (RPHM), a clinical-stage pharmaceutical company focused on the development and commercialization of therapies for ...

Mitochondrial diseases affect approximately 1 in 5,000 people worldwide, causing debilitating symptoms ranging from muscle weakness to stroke-like episodes. Some of these conditions result from ...

DURHAM, N.C.--(BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), an advanced gene editing company utilizing its novel proprietary ARCUS ® platform to develop in vivo gene editing therapies ...