Reexamining the Outcome Measures Used in Ophthalmic Gene Therapy Trials
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.
Otoferlin-Related Hearing Loss Gene Therapy DB-OTO Cleared for Clinical Trial Expansion to Spain
The clearance of the CTA is the latest in a series regulatory decisions enabling Decibel Therapeutics’ international clinical development plans.
CytoAgents’ Drug for Management of CAR-T-associated CRS Gets FDA IND Clearance
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.
Gene Therapy Voretigene Neparvovec Increases Light Sensitivity in RPE65-IRD via Peripheral Cone Function Improvements
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.
Dopamine Transporter Deficiency Syndrome Gene Therapy BGT-DTDS Gets FDA Rare Pediatric Disease Designation
Bloomsbury Genetic Therapies’ BGT-DTDS, a neuron-targeted and AAV2-mediated therapy, previously received orphan drug designation from the FDA and EC.
Around the Helix: Cell and Gene Therapy Company Updates – May 10, 2023
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Real World Data Confirm RPE65-IRD Gene Therapy Voretigene Neparvovec Efficacy in Improving Light Sensitivity
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.
Corneal Endothelial Cell Therapy Has Potential to Address Unmet Needs for Patients With Corneal Dystrophies
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.
Liso-cel Approved as Second-Line Treatment in LBCL by European Commission
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.
Systemic Lupus Erythematosus CAR-T Gets FDA Fast Track Designation
Cabaletta Bio’s CABA-201 also recently received clearance from the FDA to initiate a phase 1/2 trial.
Around the Helix: Cell and Gene Therapy Company Updates – May 3, 2023
Liso-cel Meets Primary End Point in Follicular and Mantle Cell Lymphoma Trials
Bristol Myers Squibb noted that in addition to meeting the ORR end point, both trials also met a key secondary CR rate end point.
Non–Small Cell Lung Cancer Cell Therapy Receives FDA IND Clearance
Kiromic BioPharma expects to initiate a phase 1 clinical trial for Deltacel within Q2 2023.
Luxturna Demonstrates Improvements on Multiple Vision Outcome Measures in Real-World Setting
The study found significant improvements in BCVA, in contrast to several other studies evaluating Luxturna.
GenSight Biologics’ Lumevoq Demonstrates Clinically Meaningful Improvements in Early Access Programs
The new data builds upon positive long-term follow-up data from clinical trials.
Around the Helix: Cell and Gene Therapy Company Updates – April 26, 2023
PDE6A-associated Retinitis Pigmentosa Gene Therapy May Cause Loss of Vision in Some Patients
STZ eyetrial’s treatment rAAV8.hPDE6A was otherwise deemed well-tolerated.
bluebird bio Submits BLA for Sickle Cell Gene-Edited Cell Therapy, Lovo-cel
The BLA submission comes after a series of delays which have pushed lovo-cel behind Vertex’s exa-cel in the race to approval.
Luxturna’s Safety and Efficacy Profile Supported by Retrospective Data in Patients With RPE65-Associated Inherited Retinal Dystrophy
The commercially available gene therapy, developed by Spark Therapeutics, was evaluated in multiple studies presented at ARVO’s 2023 conference.
Spark Therapeutics’ Luxturna Improves Light Sensitivity in Postmarketing Study of Inherited Retinal Dystrophy
The new positive data on the AAV based gene therapy builds upon real-world data presented at ARVO’s conference in 2022.
Therapeutic Solutions International Seeks to Expand JadiCell Treatment to Broader ARDS Population
JadiCell is already being evaluated for the treatment of COVID-19 associated ARDS in a phase 3 trial.
Lineage Tracing Sheds Light on Factors Affecting CAR-T Expansion in B-cell Malignancies
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.
Around the Helix: Cell and Gene Therapy Company Updates – April 19, 2023
Metastatic Colorectal Cancer Therapy CAR-T GCC19CART Improves Upon Third-Line SOC Therapies
Among patients treated at the higher dose level in the investigator-initiated clinical trial, the objective response rate was 50%.
CD19/20-Directed CAR-T Produces High Durable CR Rate in R/R Non-Hodgkin Lymphoma
Among 11 treated patients, 8 achieved a complete response.
News and Expert Insights in Pompe Disease Gene Therapy for International Pompe Day
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.
First Patient in Europe Dosed With B-cell Acute Lymphoblastic Leukemia CAR-T UCART22
Cellectis noted that the allogeneic product was manufactured at its new in-house facility in Paris.
Around the Helix: Cell and Gene Therapy Company Updates – April 12, 2023
Duchenne Muscular Dystrophy Gene Therapy RGX-202 Granted FDA Fast Track Designation
REGENXBIO’s RGX-202 previously received orphan drug and rare pediatric disease designations for DMD in late 2021 and early 2022, respectively.
Mesothelioma KIR-CAR T-cell Therapy Gets FDA Fast Track Designation
Verismo Therapeutics’ SynKIR-110 previously received orphan drug designation from the FDA.
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