Noah Stansfield

This is a developing story and will be updated with new information as it becomes available.

Steven W. Pipe, MD, a professor of pediatric hematology/oncology at CS Mott Children's Hospital, discussed findings from the open-label extension of the ATLAS studies at ASH 2024.

Ben Samelson-Jones, MD, PhD, the associate director of clinical in vivo gene therapy at Children's Hospital of Philadelphia, discussed follow-up data of up to 6 years with investigations of fidanacogene elaparvovec.

The trial is evaluating AURN001 for the treatment of corneal edema secondary to corneal endothelial dysfunction.

Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado, discussed 5-year follow-up data from the TRANSCEND-NHL-001 clinical trial.

Cell therapy, particularly CAR-T, is expanding into autoimmune diseases like lupus and multiple sclerosis. While promising, safety, efficacy, and broad applicability remain uncertain.

In light of the IND clearance, Arbor noted it has designed redePHine, a phase 1/2 clinical trial (NCT identifier pending) that will evaluate ABO-101 in adults and children with PH1. ABO-101 consists of mRNA expressing a novel Type V CRISPR Cas12i2 nuclease and a guide RNA directed at the human

Notably, Restem-L previously received orphan drug designation from the FDA for PM/DM in December 2024.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Tenaya Therapeutics' TN-201, an investigational adeno-associated virus (AAV)-vector based gene therapy, has been well-tolerated and shown early