The bone marrow transplant physician at St. Jude Children’s Research Hospital discussed an analysis of the sickle cell disease gene therapy he’s presenting on at ASH’s 2023 meeting.

Long-term data on lovo-cel gene therapy for sickle cell disease (SCD) reveals sustained efficacy at 60 months.

Bluebird bio’s gene therapy, marketed as Zynteglo, showed successful rates of transfusion independence up to 9 years with a reasonable safety profile in data presented at ASH 2023.

There were no treatment-related serious adverse events reported.

The FDA’s Nicole Verdun, MD; and Peter Marks, MD, PhD, offered insight on the recent approvals of exa-cel and lovo-cel, answering questions about the safety of the gene therapies and the continued collection of data on their use.

Review top news and interview highlights from the week ending December 8, 2023.

The decision was supported by efficacy data from 36 patients from the ongoing phase 1/2 HGB-206 trial (NCT02140554) and 2 patients in the phase 3 HGB-210 trial (NCT04293185).

The FDA’s decision was based on data from the CLIMB-121 and CLIMB-131 clinical trials, making it the first CRISPR-based gene therapy to be approved in the US.

The president and head of R&D at Taysha Gene Therapies discussed the early clinical evaluation of TSHA-102.

Benetic anticipates an announcement of interim results sometime around the midpoint of 2024.

The associate professor of dermatology at Stanford University discussed his experiences investigating and helping to develop Vyjuvek, approved for treating DEB.

Stempeutics mesenchymal stem cell therapy is approved in India for treating critical limb ischemia due to multiple causes.

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

The cell therapy yielded a median overall survival of around 17 months compared with a historical OS of less than 12 months.

The director of cell therapy and transplant at Penn Medicine discussed the seminar he gave at the inaugural Cell Therapy for Autoimmune Disease Summit.

ORR was 40% in the 10-mg group and 32% in the 100-mg group.

The assistant professor at MD Anderson Cancer Center discussed ongoing trials at MD Anderson and the TROP2 target for cell therapy.

For This Year in Medicine, CGTLive looks back at a landmark first approval, the new treatment opportunities it has provided, and the unmet needs in the field it has brought to light.

In observance of Crohn and Colitis Awareness Week, observed from December 1 to 7, CGTLive™ took a closer look at the clinical evaluation of the MSC-derived therapy, ExoFlo.

The director of the Adult Sickle Cell Clinic and associate professor at University of Alabama Birmingham discussed the anticipated approval of the first gene therapy for SCD.

Health Canada has greenlit a protocol amendment for Taysha Gene Therapies’ trial to include patients aged 12 years and older, rather than only adult patients.

The professor in residence of pediatrics at University of California San Francisco discussed factors that will limit the potential first approval, including access and policy.

ASC618 is designed to minimize cellular stress and may increase durability of gene therapy for hemophilia.

uniQure’s AAV9-vector therapy carrying 2 small interfering RNAs targeting GRIK2 was well-tolerated, with a hopeful risk-benefit ratio. A phase 1/2 is set to begin recruitment in late 2023.

The associate professor of ophthalmology at Harvard Medical School discussed early results from a phase 1/2 trial from Mass Eye and Ear and partners.