News

Both the second and first patient dosed in the study received the lower of the 2 doses of the gene therapy that Taysha Gene Therapies is evaluating in the phase 1/2 REVEAL trial.

The new data are from the phase 1/2 MYCHELANGELO trial of OTX-2002, an mRNA OEC therapy, in patients with hepatocellular carcinoma and other MYC-expressing tumors.

The professor of medicine and pediatrics at Washington University in St. Louis discussed future challenges and exciting research in the sarcoma field.

In observance of Rare Cancer Day, held annually on September 30, catch up on the past few months’ news and expert insights related to cell therapies in development for these indications.

Review top news and interview highlights from the week ending September 29, 2023.

Unforeseen complications may arise when treating older patients with gene therapy.

Deborah Phippard, PhD, the chief scientific officer of Precision for Medicine, discussed the history and current state of gene therapy research for neurological indications.

Atamyo has received $8.6 million in nondilutive financing from France 2030 for ATA-200's development.

The adjunct clinical assistant professor, ophthalmology, Keck School of Medicine, University of Southern California discussed new data from the ALTITTUDE trial.

Committee members were overwhelmingly against the available data supporting NurOwn as an effective treatment for mild to moderate ALS, with 17 no votes, 1 yes vote, and 1 abstention.

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

The chief scientific officer of Precision for Medicine discussed what lies on the horizon for gene therapies directed at neurological indications, such as Alzheimer disease and Parkinson disease.

Abeona has submitted a BLA for its EB-101 cell therapy.

The phase 1/2 trial is opening its second phase of recruitment after being well-tolerated in the first 4 patients and demonstrating some vision improvements.

Juan Francisco Cabello, MD, the head of the Pediatric Neurology Fellowship Program at the University of Valparaíso in Chile, discussed factors holding back more widespread adoption of newborn screening for indications treatable with gene therapy.

The associate professor at MD Anderson Cancer Center discussed the upcoming trial of NY-ESO-1 TCR/IL-15 NK cell therapy for SS and MRCLS.

Notably, the trial is assessing intravenous administration of PRGN-3005 against intraperitoneal administration in 2 separate cohorts.

NFS-05 is an ophthalmic injection that uses an AAV vector to deliver the OPA1 gene to retinal ganglion cells.

The chief scientific officer of Precision for Medicine discussed the unique aspects of clinical trials for gene therapies that need to be considered before initiation.

Krystal also noted that earlier this month the gene therapy received orphan drug designation from the FDA.

Sebastian Michels, MD, a postdoctoral fellow and member of the La Spada Lab at University of California, Irvine, discussed how his lab’s findings may open the door to developing RNA therapeutics for ALS.

The chairman, chief executive officer, and cofounder of Ocugen discussed unmet needs within retinitis pigmentosa and the potential of OCU400 gene therapy.

Review top news and interview highlights from the week ending September 22, 2023.

The FDA issued a CRL for the therapy, to be marketed as Ryoncil, in August 2023.

J. Randolph Hecht, MD, discusses the challenges researchers have faced in the development of cellular therapy for patients with solid tumors, and details how A2B530 could potentially overcome some of these obstacles.

The company is now prioritizing its gene therapy program for Rett syndrome.

The chief scientific officer of Precision for Medicine discussed the previous and current delivery methods used for gene therapies and new innovations on the horizon.

Cells from patients with ALL treated in the CARPALL study were analyzed and sequenced.