News

The director of Cell Therapy and Transplant at Penn Medicine discussed important considerations with administering CAR-T in a new field.

The endowed chair in cellular and molecular medicine at Boston Children’s Hospital discussed the rapid advancements in RNA-based treatments in the past 2 decades and potential advancements that remain on the horizon.

Bruce Cree, MD, PhD, MAS, a professor of neurology and the clinical research director of the University of California San Francisco (UCSF) Multiple Sclerosis Center discussed considerations for evaluating CAR-T in patients most likely to obtain meaningful benefit.

The chief of cardiology at Weill Cornell Medical College discussed gene therapy research into Friedreich ataxia and more.

Data from the OLE of a phase 2 clinical trial at Hadassah Medical Center were presented at the 2024 ACTRIMS meeting.

The professor from Cedars Sinai discussed how the cell therapy stands to revolutionize the treatment landscape.

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

Updated data from the RESPOND study evaluating Spinraza in patients with SMA after onasemnogene abeparvovec were presented.

The head of the early neurodevelopment clinic at Monash Children's Hospital discussed preclinical work in the context of neonatal cell therapy research.

John Ligon, MD, an assistant professor in the department of pediatrics at the University of Florida College of Medicine, discussed his team’s early findings on CAR-T and fertility from CIBMTR centers.

The associate professor of clinical pediatrics at Cincinnati Children's discussed the latest data from the phase 1/2 STAAR study of isaralgagene civaparvovec.

The gene-edited cell therapy has been approved as Lenmeldy by the FDA.

Arsa-cel, approved under the name Lenmeldy, is set to be priced at $4.25 million.

At the 2-month follow-up PET/CT scan, the patient showed a 6.6% reduction in tumor size.

The chief scientific officer of CureDuchenne discussed challenges to tackle in the field.

Bruce Cree, MD, PhD, MAS, a professor of neurology and the clinical research director of the University of California San Francisco (UCSF) Multiple Sclerosis Center discussed the potential impact of CAR-T therapy on the lives of patients with autoimmune disease.

Sandra P. Reyna, MD, the chief scientific advisor and head of global medical engagement for SMA at Novartis, discussed data from the phase 3b SMART clinical trial (NCT04851873) that were presented at MDA's 2024 conference.

In February 2024, the FDA approved a tumor-infiltrating lymphocyte therapy called lifileucel for people with advanced melanoma.

Cita-cel, approved as Carvykti, is currently under review for an indication expansion as an earlier line of therapy, which the FDA will decide on by April 5, 2024.

Review top news and interview highlights from the week ending March 15, 2024.

Breyanzi was approved for the expanded indication under the accelerated approval pathway.

The director of the Manton Center for Orphan Disease Research at Boston Children’s Hospital discussed takeaways from a session on rare disease gene therapy at MDA.

bluebird bio stated that it is working on additional agreements with more than 15 more Medicaid agencies.

Investigators found AGTC-501 to have a favorable benefit-risk profile.

The lead scientist at Percheron Therapeutics discussed the design of the study assessing proteomics of ATL1102.

Bruce Cree, MD, PhD, MAS, a professor of neurology and the clinical research director of the University of California San Francisco (UCSF) Multiple Sclerosis Center discussed the need for randomized-control trials for CAR-T in lupus and other autoimmune diseases.

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

Verve Therapeutics plans to expand evaluations into the US after an IND clearance was delayed by a clinical hold.