CSPC Pharmaceutical Group Ltd. has gained clinical trial approval from China’s National Medical Products Administration (NMPA) for SYH-2062 injection, a double-stranded small interfering RNA (siRNA) drug to treat hypertension.
After raising AU$40 million (US$25.9 million) earlier this year to advance lead candidate VP-001 for treating retinitis pigmentosa type 11, PYC Therapeutics is now advancing PYC-003 to the clinic for polycystic kidney disease.
In the largest collaboration of 2024, Sarepta Therapeutics Inc. and Arrowhead Pharmaceuticals Inc. entered a sprawling global licensing deal that includes a swath of clinical and preclinical candidates targeting rare genetic diseases. Under the terms, Sarepta gains access to existing and potential future compounds derived from the RNAi platform developed by Arrowhead, with the latter eligible for payments potentially exceeding $11 billion.
