SMN1 replacement using AAV9 has improved survival and motor outcomes in spinal muscular atrophy (SMA), but inefficient CNS delivery necessitates high systemic doses, potentially narrowing the ...

Shanghai Henlius Biotech Inc.’s HLX-109, an anti-IL1RAP monoclonal antibody, has received approval from the Human Research ...

Abbvie Inc. is paying Nxera Pharma Co. Ltd. $100 million up front for worldwide rights to NXE-0033744, a phase II-ready oral EP4 receptor agonist designed to act locally in the gastrointestinal tract ...

T-Therapeutics Ltd. has raised $133 million in an oversubscribed series B, providing the means to deliver clinical proof of concept for its T-cell receptor-CD3 bispecifics in solid tumors and ...

A pair of popular modalities stands at the back-end-loaded, potential $7 billion-plus deal inked by Boehringer Ingelheim GmbH ...

For only the fourth time since World War II, the U.S. midterm election flipped control of both the House and the Senate. (The ...

Alethio Therapeutics Ltd. has closed a £67 million (US$90.3 million) series A round that with a fair wind will see its lead ...

Rheumatoid arthritis (RA) and osteoarthritis (OA) are prevalent joint disorders that differ in etiology but share chronic inflammation, synovitis and progressive cartilage degradation. Using mRNA ...

Blueprint Medicines Corp. has discovered new proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase-binding moiety covalently linked to a cyclin-dependent kinase 4 ...

Kimia Therapeutics Inc. has signed a clinical collaboration agreement with Cstone Pharmaceuticals Co. Ltd. to advance KT-721 into phase I development.

Suzhou Puhe Biopharma Co. Ltd. has prepared and tested new S-adenosylmethionine synthase isoform type-2 (Mat2A) inhibitors potentially useful for the treatment of cancer.

Convalife Pharmaceuticals Co. Ltd. has divulged new diphenyl ether derivatives acting as thyroid hormone receptor β (THRβ) agonists potentially useful for the treatment of alopecia.