Noah Stansfield

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.

Sattar Khoshkhoo, MD, the founding director of the Epilepsy Genetics Clinic at Brigham and Women's Hospital, discussed his nonclinical research on disease pathogenesis in MTLE.

Novartis stated that the decision was made in light of a recommendation from an independent data monitoring committee.

The company also announced its plans for a modified study protocol that may allow a roadway to approval.

Study author Mya C. Schiess, MD, commented on trends seen in the blinded findings so far.

ATA-100 is currently being evaluated in a multicenter phase 1/2 clinical trial (NCT05224505) in Denmark, France, and United Kingdom.

In light of the IND clearance, uniQure announced its intention to carry out a phase 1/2a clinical trial, with screening of potential participants with refractory MTLE anticipated to begin in the last 3 months of 2023.

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

The therapy is expected to be able to treat both the lung and liver manifestations of AATD.

The data come from 30 patients treated across 2 clinical trials and 9 patients treated in expanded access frameworks who had follow-up times ranging from 0.64 years to 12.19 years.

Acclaim-1, Acclaim-2, and Acclaim-3 will evaluate Reqorsa in combination with osimertinib, pembrolizumab, and atezolizumab, respectively.

It was the first time the Eye Center treated a patient with a gene therapy.

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

Adrian Kilcoyne, MD, MPH, MBA, the chief medical officer of Celularity, discussed what the company learned from relitigating results of legacy trials for a mesenchymal stem cell therapy in Crohn disease.

ATSN-201 utilizes Atsena’s novel spreading capsid AAV.SPR, which is expected to spread laterally and allow transduction of the gene therapy in the central retina.

The FDA’s decision was based on data from the randomized, open-label phase 3 COMMANDS clinical trial.