Adverse genetic mutations can cause harm and are due to various circumstances. "Jumping genes" are one cause of mutations, but cells try and combat them with a specialized RNA called piRNA.

Setmelanotide is a melanocortin-4 receptor (MC4R) agonist designed to treat hyperphagia and obesity. Rhythm is evaluating setmelanotide in a global Phase 3 trial in patients with acquired hypothalamic ...

Arbor Biotechnologies has closed $73.9m for its gene editing therapeutic pipeline aimed at liver and central nervous system ...

Dr Anup Rawool, Consultant in Clinical and Cancer Genetics at Sahyadri Hospitals, Pune, discusses innovative therapies and ...

Announcement follows FDA's February 2025 decision to grantradiprodil Breakthrough Therapy designation Company remains on track to initiate Phase 3 pivotal trial ofradiprodil ...

UCART22 Phase 1 dataset and late-stage development strategy expected in the third quarter of 2025; Orphan Drug Designation (ODD) and Rare ...

The FDA granted fast track designation to ATSN-201 for the treatment of X-linked retinoschisis, according to a press release ...

a leading biopharmaceutical company for cell and gene therapy CDMO and novel drug development, announced today that its investigational drug EN001 has been granted Orphan Drug Designation (ODD ...

A disease is classed as rare when it affects fewer than one in 2,000 people. New orphan drugs can provide rare disease patients with hope for a better quality of life when all existing treatment ...

MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) announced that the European Commission has granted Advanced Therapy Medicinal ...