There were no serious adverse events or treatment-related mortalities. Evidence Rating Level: 2 (Good) Study Rundown: Transfusion-dependent β-thalassemia (TDT) is a condition that results in iron ...

Thirty patients have received the gene therapy within a clinical trial and early-access program, and the foundation also hopes to seek approval in the US.

Oxford, UK – 5 February 2025: OXB (LSE: OXB), a quality and innovation-led cell and gene therapy CDMO, today announces the appointment of Jefferies International Limited ("Jefferies") as its Joint ...

The cuts at two Massachusetts plants follow further site closures and layoffs across the company over the past few years.

Telethon Foundation is seeking approval for a gene therapy to treat the rare disease Wiskott-Aldrich syndrome.

DELRAY BEACH, FL / ACCESS Newswire / February 4, 2025 / Adolore BioTherapeutics ("Adolore" or the "Company"), is developing ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three-to-six times each week.

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...

Despite recent evidence of its financial turnaround, Thermo Fisher’s layoff spree will continue into 2025. | Despite recent ...

Potent in vivo gene editing in skeletal muscle of non-human primates by a novel, ultracompact CRISPR system delivered via a single AAV vector.

Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of ...