Noah Stansfield

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.

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 decision, which aligns with its indication in the US, was based on efficacy and safety results from Bristol Myers Squibb’s TRANSFORM clinical trial in forms of large B-cell lymphoma.

Cabaletta Bio’s CABA-201 also recently received clearance from the FDA to initiate a phase 1/2 trial.

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

Bristol Myers Squibb noted that in addition to meeting the ORR end point, both trials also met a key secondary CR rate end point.

Kiromic BioPharma expects to initiate a phase 1 clinical trial for Deltacel within Q2 2023.

The study found significant improvements in BCVA, in contrast to several other studies evaluating Luxturna.

The new data builds upon positive long-term follow-up data from clinical trials.

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

STZ eyetrial’s treatment rAAV8.hPDE6A was otherwise deemed well-tolerated.

The BLA submission comes after a series of delays which have pushed lovo-cel behind Vertex’s exa-cel in the race to approval.

The commercially available gene therapy, developed by Spark Therapeutics, was evaluated in multiple studies presented at ARVO’s 2023 conference.

The new positive data on the AAV based gene therapy builds upon real-world data presented at ARVO’s conference in 2022.

JadiCell is already being evaluated for the treatment of COVID-19 associated ARDS in a phase 3 trial.

The investigators created the largest known CAR-T atlas, with more than 800,000 cells in total, providing a new exploration into the phenotyping of T-cells.

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

Among patients treated at the higher dose level in the investigator-initiated clinical trial, the objective response rate was 50%.

Among 11 treated patients, 8 achieved a complete response.

In observance of International Pompe Day, held annually on April 15, catch up on the past year’s news and expert insights related to gene therapies in development for this rare disease.

Cellectis noted that the allogeneic product was manufactured at its new in-house facility in Paris.

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

REGENXBIO’s RGX-202 previously received orphan drug and rare pediatric disease designations for DMD in late 2021 and early 2022, respectively.

Verismo Therapeutics’ SynKIR-110 previously received orphan drug designation from the FDA.

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

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

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

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

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.

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