Noah Stansfield

Deborah Phippard, PhD, the chief scientific officer of Precision for Medicine, discussed what may be next for the field of gene therapy after early successes in single-mutation disorders.

In September 2023, LEU011 was granted an Innovation Passport for the treatment of solid tumors expressing NKG2D ligands.

Deborah Phippard, PhD, 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.

The patient received the trial’s low dose of TN-201 at the Cleveland Clinic's Hypertrophic Cardiomyopathy Center, in Cleveland, Ohio.

The move was carried out in accordance with a recommendation for dose-escalation made by the trial’s independent Data Safety Monitoring Committee.

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

The company stated that based on the meeting, the trial’s primary end points will remain the same.

The company also announced it has onboarded 15 transplant centers in the United States for the use of omidubicel, thus exceeding the goal it had set for itself for 2023.

Sponsors who hope to participate will be able to apply from January 2 to March 1, 2024.

Notably, no patients experienced cases of immune effector cell-associated neurotoxicity syndrome.

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.

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.

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.

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.

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.

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

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

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

Among 12 patients evaluable for efficacy, 10 patients experienced stabilization or improvement from baseline in BCVA in their treated eyes.

Juan Francisco Cabello, MD, the head of the Pediatric Neurology Fellowship Program at the University of Valparaíso in Chile, discussed the current state of newborn screening and what needs to change.

The PDUFA data has been set for March 18, 2024.

The companies plan to evaluate the combination therapy in patients with HL in the phase 2 LuminICE-203 clinical trial.

The study will utilize a pediatric safety run-in that will include 2 patients.

Sattar Khoshkhoo, MD, the founding director of the Epilepsy Genetics Clinic at Brigham and Women's Hospital, spoke about how his lab’s findings may imply a need to rethink epilepsy treatment development.

Kyverna stated that the patient, who received KYV-101 in July at the University of Colorado Anschutz Medical Campus, did not experience any ICANS.

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