JCR Pharmaceuticals has signed a licence agreement with Alexion, AstraZeneca Rare Disease, for its proprietary JUST-AAV capsids.

JCR Pharma signs agreement with Alexion for proprietary JUST-AAV capsids to develop genomic medicines: Hyogo, Japan Thursday, July 10, 2025, 10:00 Hrs [IST] JCR Pharmaceuticals Co ...

Merck & Co. Inc. has strengthened its cardiopulmonary pipeline with its $10 billion acquisition of London-based-Verona Pharma plc. The deal brings Merck Ohtuvayre (ensifentrine) for chronic ...

In an opinion article, investigators argue that gene therapies may not achieve their full potential if the innate inflammation in Duchenne muscular dystrophy is not effectively managed.

SGT-501 is a novel gene therapy for rare, life-threatening, genetic arrhythmogenic disease with no approved therapies - - SGT-501 has received Orphan Drug Designation and Rare Pediatric Disease Design ...

Study met its primary and key secondary endpoints, demonstrating rapid and sustained improvement in near visual acuity; Safety profile consistent with previous clinical trials and ...

JCR announced that it has entered into a license agreement with Alexion, for JCR’s new, proprietary JUST-AAV capsids to ...

Monoclonal antibodies show strong therapeutic potential against neurodegenerative diseases, but a major challenge is ensuring ...

The conversion of acetylcholine binding into ion conduction across the membrane is becoming more clearly understood in terms of the structure of the receptor and its transitions. A high-resolution ...

Seropositivity for antibodies that bind to or block ganglionic acetylcholine receptors identifies patients with various forms of autoimmune autonomic neuropathy and distinguishes these disorders ...