Hematology

CD19-directed CAR-T-cell therapy resulted in sustained remission for over a year and no return of autoimmunity for patients with systemic lupus erythematosus, idiopathic inflammatory myositis, and systemic sclerosis.

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.

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

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.

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.

Review top news and interview highlights from the week ending December 1, 2023.

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

The company also plans to submit an MAA to the EMA in the first half of 2024.

The FDA has received reports of multiple T-cell malignancies in patients after approved CAR-T treatment.

The CAR-T was already being assessed in the ongoing phase 1a/1b NEXICART-1 clinical trial in Israel ahead of this IND clearance by the FDA.

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

The date for the AdComm and the new PDUFA date are yet to be announced.

Review top news and interview highlights from the week ending November 17, 2023.

Vertex and CRISPR’s Exa-cel, which remains an investigational treatment in the United States, is the first CRISPR-based gene therapy to be approved in the UK, where it will be marketed as Casgevy.

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

Review top news and interview highlights from the week ending November 10, 2023.

The therapy was previously granted priority review in May 2023.

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

David Rawlings, MD, the director of the Center for Immunity and Immunotherapies at Seattle Children's Research Institute, discussed findings from several preclinical studies that could help push engineered B-cell therapies towards clinical trials.

Review top news and interview highlights from the week ending Novmber 3, 2023.

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

The members of the AdComm were generally of the opinion that the off-target analyses conducted by Vertex were reasonably robust.