Noah Stansfield

There were no serious adverse events reported and no patients discontinued from the study.

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

The findings, presented at ASGCT’s 2023 conference, included an analysis of biomarker data in relation to disease severity.

All 8 patients with light chain amyloidosis who received HBI0101 responded.

Following implantation with CellGenTech’s LCAT-GMAC, the patient’s serum LCAT activity increased by approximately 50% of baseline.

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

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.

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.

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.

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

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

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.