FDA Accepts IND Application for Cystic Fibrosis Gene Therapy KB407
The phase 1 clinical trial from Krystal Biotech is expected to begin this year.
HIV Gene Therapies Continue to Move Through the Pipeline With Promise
Seven participants have been dosed with AGT103-T, an autologous T-cell therapy currently being evaluated in a phase 1 trial.
COVID-19 Cell Therapy Trial Dosing Complete With Good Safety Profile
Elderly or high-risk patients with COVID-19 received 1 of 4 escalating dose levels.
Clinical Hold Lifted on Celyad's Phase 1b Trial in Refractory Metastatic Colorectal Cancer
The company voluntarily paused the trial in February 2022 to investigate 2 reported deaths in the trial.
James Beck, PhD, on Laying Groundwork for Gene Therapy in Parkinson Disease
The chief scientific officer and senior vice president of the Parkinson’s Foundation discussed remaining research and education needs to support the future of genetic medicines.
Gene Therapy: The Future of Rare Disease Treatment
Experts shared opinions about the future of gene therapy in sickle cell and other rare diseases.
CGTLive’s Weekly Rewind – July 29, 2022
Review top news and interview highlights from the week ending July 29, 2022.
Sarepta Planning BLA Submission of DMD Gene Therapy
The announcement follows positive data updates from the ENDEAVOR and SRP-9001-101 studies.
Mazyar Shadman, MD, MPH, on Addressing Unmet Needs in Follicular Lymphoma With MB-106
The associate professor at Fred Hutch Cancer Center discussed next steps with the clinical trial being conducted Fred Hutch Cancer Center.
Natural Killer Cell Therapy Trial Doses First Patient With NSCLC
CYTO-102 is being developed by City of Hope and CytoImmune.
Editas' Gene Therapy Trial for Sickle Cell Initiates After Hold Lifted
The company announced successful neutrophil and platelet engraftment for the first patient dosed.
Nilanjan Ghosh, MD, PhD, on New Avenues of CAR T Research in Lymphoma
The chief of the lymphoma division and oncologist at Levine Cancer Institute discussed new CAR T-cell therapies being investigated for hematologic malignancies.
Gastric/Gastroesophageal Junction Cell Therapy Trial Doses First Patient
CYNK-101 has previously been granted fast track and orphan drug designation.
Around the Helix: Cell and Gene Therapy Company Updates – July 27, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
First Patient Dosed in RAG1-SCID Gene Therapy Trial
Mustang Bio plans to add more clinical trial sites in the near future.
Liso-Cel Approval Expands CAR T-Cell Access in LBCL
Nilanjan Ghosh, MD, PhD, discussed the impact of liso-cel's approval.
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.
