The global orphan drug market size was USD 154.20 Billion in 2022 and is expected to register a rapid revenue CAGR of 12.3 % ...

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 ...

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 ...

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

The gene therapy world is in turmoil, but Arbor, armed with more than a billion dollars in partnerships and raises, is going forward.

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

Healthcare experts advocate genetic counselling, family tree mapping, and incentives for indigenous drug production to address rare diseases.

Data Safety Monitoring Board (DSMB) recommendation to initiate second cohort follows review of Phase 1/Phase 2 LION-CS101 ...

FDA granted priority review to Company's BLA for PRGN-2012 for the treatment of adults with recurrent respiratory papillomatosis and set PDUFA target action date ...

Korro Bio, Inc. (Korro) (Nasdaq: KRRO), a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on editing RNA for both rare and highly prevalent ...