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The company’s pipeline is down to 1 remaining clinical-stage program that is enrolling in its first-in-human trial.

The director of the center for gene therapy at Nationwide Children's Hospital discussed the prospect of gene therapy approvals in Duchenne muscular dystrophy and the associated challenges that remain to be addressed.

Peter Marks, MD, PhD, director, Center for Biologics Evaluation and Research, FDA, discussed his keynote address at the 2023 MDA Conference. c

Review top news and interview highlights from the week ending April 7, 2023.

The trial’s dose-escalation cohorts are assessing results from patients with type 2a and type 1 diseases separately.

The locum consultant of pediatric BMT and leukemia at Royal Manchester Children’s Hospital discussed the center’s experience in delivering the approved gene therapy to children with MLD.

Study authors Matthew Charman, PhD, and Matthew D. Weitzman, PhD, elaborated on the findings and implications of their research.

Although the TCB-202-001 trial ended prematurely due to the COVID-19 pandemic, promising results warrant a phase 2 trial.

The spine surgeon from The Orthopedic Center of St. Louis discussed recent data on IDCT from the phase 1/2 DGX-A01 study.

Investigator Matthew Charman, PhD, told CGTLive™ that the new knowledge of the viral replication process could potentially inform the development of future gene therapies.

The professor and director of the Sen. Paul D. Wellstone Muscular Dystrophy Specialized Research Center at University of Washington School of Medicine discussed the PDC group and collaborating with advocacy groups.

No treatment-emergent adverse events experienced were deemed related to AstroRx, although 9 serious TEAEs did occur.

Atsena Therapeutics’ AAV gene therapy is being evaluated in 15 patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D across 5 cohorts of various doses. Additional data are expected to be presented later this year.

The neurology resident at Penn Medicine spoke about the preparations taken in the lead up to tofersen’s PDUFA date, which is set for April 25, 2023.

The clinical trial of HG004 for RPE65-LCA2 dosed its first patient in February 2023.

The rolling BLAs also included requests for priority review of the CRISPR/Cas9 gene-edited therapy, and is supported by data from the phase 3 CLIMB-111 and CLIMB-121 studies, as well as the long-term follow-up study CLIMB-131.

The co-CEO of BrainStorm Cell Therapeutics spoke about the current standards of care in ALS and the potential of NurOwn to address unmet needs.

The company also recently announced updates in the MATRICS-1 clinical trial for hemorrhagic trauma.

Precigen’s investigational UltraCAR-T therapy is being assessed in a dose-escalation and -expansion study that will include patients with solid tumors, chronic lymphocytic leukemia, mantle cell lymphoma, acute lymphoblastic leukemia, and diffuse large B-cell lymphoma.

Review top news and interview highlights from the week ending March 31, 2023.

CB-011 and CB-010 are being evaluated for the treatment of r/r multiple myeloma and r/r B-cell non-Hodgkin lymphoma, respectively.

The associate professor of neurology at Columbia University spoke about the importance of identifying patients with ALS-associated gene mutations as early as possible.

Most patients with RP treated with MCO-010 had clinically meaningful improvements in vision.

The Study Safety Committee has recommended that the trial proceed to enroll patients in the highest-dose cohort.

The Lillehei professor in stem cell and regenerative cardiovascular medicine at the University of Minnesota spoke about preclinical research with an iPS cell-derived product.

The gene-edited cell therapy had previously been under a partial clinical hold restricting pediatric enrollment to the clinical trial.

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

Intellia Therapeutics received clearance to enroll patients from the US in the global phase 2 portion of its clinical trial earlier this month.