Susan Ruediger on Partnering to Develop CMT Gene Therapies
Susan Ruediger, founder and chief mission officer, CMT Research Foundation, discussed a new partnership with Nationwide Children’s Hospital.
FDA Clears IND Application for TIL Therapy OBX-115 for R/R Metastatic Melanoma
In preclinical studies, OBX-115 demonstrated improved TIL persistence, potency, and tumor control compared with unengineered TILs.
Nanoscope Gene Therapy Continues Progress in Stargardt Disease
Data is expected in the first quarter of 2023 for both Stargardt disease and retinitis pigmentosa.
Steven W. Pipe, MD, on Conducting Further Research in Hemophilia
The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed current studies evaluating gene therapies in hemophilia.
Patient Dosed With Mesenchymal Stem Cell-Derived Therapy for Osteoarthritis
Reported efficacy data included a WOMAC score reduction at 6 months following a single dose.
Gene Therapy Shows Durable Bilateral Improvements in Leber Hereditary Optical Neuropathy
The long-term RESTORE study included data from 55 participants in previous phase 3 trials.
Hemophilia B Gene Therapy Shows Long-Term Ability to Reduce FIX Consumption
Most patients treated with FLT180a had elevated FIX levels over 2 years after treatment.
Peter Bross, MD, on the Importance of Transparency and Communication With the FDA
The chief of the oncology branch of the Center for Biologics Evaluation and Research at the FDA discussed how academia and industry can work best with regulatory agencies.
Phase 3 Cardiosphere-Derived Cell Therapy Trial Doses First Patient With DMD
Recent long-term data from the phase 2 extension has also demonstrated CAP-1002's efficacy in Duchenne.
CGTLive’s Weekly Rewind – July 22, 2022
Review top news and interview highlights from the week ending July 22, 2022.
Upstaza Approved by European Commission for AADC Deficiency
The gene therapy received a positive CHMP opinion in May 2022.
Paul Wuh-Liang Hwu, MD, PhD, on Questions Remaining With AADC Deficiency
Upstaza has been granted marketing authorization by the European Commission.
Renovacor and University of Utah Initiate Gene Therapy Research Program for Arrhythmogenic Cardiomyopathy
Robin Shaw, MD, PhD, Professor of Medicine at the University of Utah and Director of the Nora Eccles Harrison Cardiovascular Research and Training Institute, shared his thoughts on the program.
Allogeneic CAR T-Cell Therapy Recognized in T-Cell Malignancies
WU-CART-007 has been granted fast track, rare pediatric disease, and orphan drug designations.
Craig M. McDonald, MD, on Developing Gene Therapy for Different Populations of DMD
The principal investigator of the HOPE-2 trial discussed results of the trial in the non-ambulatory population of patients with Duchenne muscular dystrophy.
Around the Helix: Cell and Gene Therapy Company Updates – July 20, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Gene Therapy Fails to Improve Survival in Platinum-Resistant Ovarian Cancer
Ofra-vec continues to be evaluated in phase 2 trials for glioblastoma and colorectal cancer.
Richard Fessler, MD, on Evaluating Oligodendrocyte Cell Therapy for Spinal Cord Injury
The professor of neurosurgery at Rush University Medical School discussed data seen in the phase 1/2 clinical trial of LCTOPC1.
Angelman Syndrome Gene Therapy Shows Promising Efficacy in Higher-Dose Cohorts
Ultragenyx also exercised its option to acquire GeneTx, its partner in developing GTX-102.
Cell Therapy Trial Fails Primary Endpoint in Treating Muscle Injury After Arthoplasty for Hip Fracture
PLX-PAD is also being studied for the treatment of steroid-refractory chronic graft versus host disease.
Wolfgang Miesbach, MD, PhD, on Future Research with EtranaDez
The professor and head of coagulation disorders and Comprehensive Care Centre, University Hospital of Frankfurt, Germany, discussed future integration of EtranaDez into hemophilia B treatment.
Challenges With Future Stem Cell Therapies in Parkinson Disease
Rajesh Pahwa, MD, FANA, FAAN, discussed balancing hope with current challenges with stem cell approaches in PD.
Daniel Judge, MD, on Translational Gene Therapy for Cardiac Disorders
Genetics is the future of heart failure and cardiomyopathy treatment, Judge told CGTLive in an interview.
Stephanie Cherqui, PhD, on Bringing Attention to Cystinosis and Gene Therapy
The associate professor from UC San Diego discussed promising efficacy and safety data from a phase 1/2 trial.
WVU Cancer Institute Cellular Therapy Program Recognized by the Foundation for the Accreditation of Cellular Therapy
Lauren Veltri, MD Director, WVU Cancer Institute Hematopoietic Malignancy and Cellular Therapy Program, discussed the recent FACT accreditation.
CGTLive’s Weekly Rewind – July 15, 2022
Review top news and interview highlights from the week ending July 15, 2022.
Bone to Optimize Phase 2b Trial of High-Risk Tibial Fracture Cell Therapy
The adjustments come after the company announced a reprioritization to focus on ALLOB in September 2021.
ATA188 Shows No Significant Improvements in MS, Study to Continue
There were not enough data at 6 months to predict expanded disability status scale (EDSS) improvements after treatment.
MPSII Gene Therapy Granted Orphan Drug Designation
AVROBIO decided to deprioritize their Fabry disease gene therapy program in January 2022.
Ixo-Vec Back in Phase 2 Trials, Now for Wet Age-Related Macular Degeneration
After submitting an amended IND, Adverum’s gene therapy is set to be evaluated in the phase 2 LUNA trial.
