Key opportunities in the gene therapy market include treating genetic disorders and cancer, driven by rising FDA approvals and biotechnological advancements. The in vivo segment, cancer treatments, ...

Few companies stand better poised than Beam Therapeutics to reap the first fruits of the FDA’s promised flexibility toward cell and gene therapies. | Riding a regulatory win for its base editing ...

The global cell and gene therapy market is experiencing explosive growth. Key growth factors include rising chronic/genetic disease prevalence, advancements in gene editing (CRISPR) and viral vectors, ...

The Director of the San Raffaele-Telethon Institute for Gene Therapy Luigi Naldini reflects on what a recent Nature study reveals and about the future of in vivo gene transfer “Our study provides an ...

While the clinical hold has been lifted on the MAGNITUDE-2 trial, it remains in place on the MAGNITUDE study.

A team of scientists from the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy, has identified a unique window shortly after birth in which circulating blood stem cells can ...

GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia.

Recordati Joins Boehringer Ingelheim Venture Fund in Financing Round, with Participation from Delos and Blue Bay Capital New Investment, Combined with Recent $8.4 Million ARPA-H Award, Brings Funds ...

A 24‑week pregnant woman fears for her unborn baby, who is developing with a sacrococcygeal teratoma so large and vascularized that it nearly surpasses the size of the fetus itself. Faced with this ...

The illustration shows how high trafficking of blood stem cells in the circulation early post-natally allows lentiviral-vector mediated in vivo gene transfer. “After birth, blood stem cells need to ...