A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists from St. Jude Children's Research Hospital and University College London ...

Gregory Russotti is the Chief Technology and Manufacturing Officer at Century Therapeutics, a company developing iPSC-derived, allogeneic immune cell therapy products for hematology/oncology ...

A single intravenous dose of etranacogene dezaparvovec gene therapy maintained factor IX (FIX) activity at 45.7 IU/dL after 5 years in patients with hemophilia B, eliminating the need for prophylaxis ...

Infant KJ Muldoon was born with carbamoyl phosphate synthetase 1 (CPS1) deficiency, a condition so rare that only about 1 in a million babies are diagnosed with it. Outcomes are often severe, ...

A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists from St. Jude Children's Research Hospital and University College London ...

Within minutes of KJ Muldoon’s birth, doctors knew there was something very wrong. Five weeks premature, his little arms went rigid when lifted and shook oddly on the way back down. An attentive ...

Forbes contributors publish independent expert analyses and insights. William A. Haseltine, Ph.D., covers genomics and regenerative medicine Today, the world stands at a crossroads in genetic medicine ...

The company has made more than 50 deals for its proprietary technology as it looks forward to a potentially “transformative” ...

BERWYN, Pa., July 29, 2025 (GLOBE NEWSWIRE) -- AscellaHealth, a global partner delivering customizable solutions to support the specialty pharmaceutical industry, highlights the value of its HUB ...

Elaine Chen covers biotech, co-writes The Readout newsletter, and co-hosts STAT’s weekly biotech podcast, The Readout Loud. You can reach Elaine on Signal at elaineywchen.70. Adam Feuerstein is a ...

17 client-owned dogs with radiographically confirmed OA received 1 mg intramuscular ElenaVet™ once a week for 10 weeks. Pain Severity Score (PSS) fell from 5.25 → 3.25 and Pain Interference Score (PIS ...