News

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

Among 27 evaluated patients with relapsed/refractory B-cell acute lymphoblastic leukemia, 18 achieved a complete response.

The professor at Ohio State University and University of California – San Francisco discussed updated data from a phase 1 study of GDNF gene therapy in patients with PD.

Updated data from a phase 1 study of PCXR201, formerly known as Flexion’s FX201, were presented at the 2023 ASCGT Annual Meeting.

Vitalgen’s gene therapy was generally well-tolerated with no serious adverse events reported.

Bart P. Leroy, MD, PhD, head of the Department of Ophthalmology at the Center for Medical Genetics of Ghent University Hospital, discussed the need to reevaluate the goal posts used to determine whether gene therapies for eye indications come to market.

The clearance of the CTA is the latest in a series regulatory decisions enabling Decibel Therapeutics’ international clinical development plans.

The cofounder and chief scientific officer of Xcell Biosciences discussed the company’s efforts to introduce new technologies for improving cell therapy development and manufacturing.

CTO1681, which is designed to lower NF-kB signaling without eliminating it altogether, was previously evaluated for safety in a phase 1 clinical trial in healthy adults.

The assistant professor of stem cell transplantation at MD Anderson Cancer Center discussed positive data in ccRCC that he believes should excite the solid tumor field as a whole.

Bart P. Leroy, MD, PhD, head of the Department of Ophthalmology at the Center for Medical Genetics of Ghent University Hospital, discussed new real world data regarding Spark Therapeutics’ Luxturna that was presented at ARVO’s 2023 conference.

The chief medical officer at Atsena Therapeutics discussed updated, 6-month data from a phase 1/2 trial presented at ARVO 2023.

Committee members were mostly split, ultimately voting 8–6 in favor of the benefit-risk profile of SRP-9001 supporting accelerated approval.

Review top news and interview highlights from the week ending May 12, 2023.

The director of therapeutic genome engineering, St. Jude Children’s Research Hospital discussed recent research published in Nature Biomedical Engineering.

Bloomsbury Genetic Therapies’ BGT-DTDS, a neuron-targeted and AAV2-mediated therapy, previously received orphan drug designation from the FDA and EC.

The director of clinical research at Sierra Eye Associates discussed 4D Molecular Therapeutics’ plans for 4D-150 in both wet AMD and diabetic macular edema.

The neoantigen-specific TCR therapy is the first to enter clinical trials out of a collaboration with Genentech.

The cofounder and chief scientific officer of Xcell Biosciences discussed the company’s manufacturing-focused approach to overcoming the challenges of the solid tumor microenvironment.

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

Biogen and Novartis terminated collaborations with the company that first initiated in 2020.

The cell therapy field applications staff scientist at ThermoFisher discussed advantages of the company’s DynaCellect system.

Bart P. Leroy, MD, PhD, head, Department of Ophthalmology, Center for Medical Genetics, Ghent University Hospital, discussed the ongoing efforts to verify the efficacy of Spark Therapeutics’ Luxturna.

SynKIR-110 was also granted fast track designation by the FDA in April 2023.

The director of clinical research at Sierra Eye Associates discussed the latest results from a clinical trial evaluating 4D Molecular Therapeutics’ gene therapy, 4D-150, that were presented at ARVO’s 2023 conference.

The single-administration allogeneic cell therapy has shown positive safety—and signs of efficacy—in preliminary data from the first 2 patients dosed with the Neurona Therapeutics’ product.

Arnaud Lacoste, PhD, the chief scientific officer of Aurion Biotech, discussed AURN001, a corneal endothelial cell therapy that was recently approved in Japan, and had a nonclinical data read out at ARVO 2023.

The head of the Department of Ophthalmology at the Center for Medical Genetics at Ghent University Hospital discussed the need to reevaluate requirements for regulatory approval for ophthalmology gene therapies.

Krystal Biotech's B-VEC was well-tolerated and the treated patient experienced significant improvement in visual acuity.

CK0803 is being evaluated for safety and tolerability in the REGALS study.