The Potential of Gene Therapy for AADC Deficiency: Paul Wuh-Liang Hwu, MD, PhD
The professor from National Taiwan University Hospital discussed the mechanism of gene therapy in AADC deficiency.
FDA Grant Supports Recessive Dystrophic Epidermolysis Bullosa Gene Therapy Study
Dabocemagene autoficel has previously received orphan drug, rare pediatric disease, fast track, and regenerative medicine advanced therapy designations.
Encapsulated Cell Therapy for Retinal Diseases
John Pollack, MD, discusses gene therapy advancements for retinal diseases.
Developing Personalized Immunotherapies for Solid Tumors
Jessica Baker Flechtner, PhD, chief scientific officer, Genocea, discussed the company’s focus on solid tumors and manufacturing cell therapies.
Gene Editing Clinical Trial for PKU Announced
Homology is also developing a gene therapy, HMI-102, being evaluated in the phase 1/2 pheNIX trial.
Improving Practicality of Cell and Gene Therapies
Josh Ludwig, global director, commercial operations, ScaleReady, discussed making cell and gene therapy widely practical and viable for patients with cancer.
Gene Therapy Promotes Wound Closure of Diabetic Foot Ulcers
VM202 was found to be particularly effective in treating neuroischemic ulcers.
Collaborating to Develop Oncologic Cell Therapies
André Choulika, PhD, chief executive officer and cofounder, Cellectis, discussed the company’s partnerships and science.
Eli-cel Gene Therapy for Early CALD to Be Withdrawn From EU, UK
bluebird bio is also planning to withdraw the marketing authorization for their β-thalassemia therapy, beti-cel, from the EU and UK.
Mantle Cell Lymphoma Treatment Landscape 1 Year After CAR T Approval
Michael Wang, MD, discussed the evolution of treatment in the MCL space specifically with the emergence of chimeric antigen receptor T cells.
The Potential of ALLO-715 in Relapsed/Refractory Multiple Myeloma: Doris Hansen, MD
The hematologist from Moffitt Cancer Center discussed the promise of ALLO-715 in relapsed/refractory multiple myeloma.
Next Steps With CAR T-Cell Therapy in Multiple Myeloma: Omar Castaneda Puglianini, MD
The hematologist and medical oncologist discussed next steps with CAR T-cell therapy in penta-refractory multiple myeloma.
In-Vivo Genome Editing Therapy Shows Efficacy in Children With MMA
The phase 1/2 SUNRISE trial is now enrolling patients as young as 6 months old with methylmalonic acidemia after positive review under a DSMB.
Potential of mRNA-Based Gene Therapies
Jake Becraft, PhD, chief executive officer and cofounder, Strand Therapeutics, discussed the company’s future research and plans.
GeneTherapyLive’s Weekly Rewind – October 22, 2021
Review top news and interview highlights from the week ending October 22, 2021.
Developing Customizable CAR T-Cell Therapies
Rick Fair, president and chief executive officer, Bellicum Pharmaceuticals, discussed the company’s GoCAR, iMC, and caspaCIDe technologies.
Dual-Targeted CAR T Therapy Efficacious in Relapsed/Refractory B-Cell Malignancies
The phase 1 study met its primary endpoints, with 97% of the CAR T-cell therapies reaching the protocol-specified dose and no dose-limiting toxicities during dose escalation.
Gene Therapy for LAD-1 Demonstrates Efficacy
Rocket Pharmaceuticals announced positive data on RP-L201 in leukocyte adhesion deficiency-I, as well as RP-A501 for Danon disease.
Gene Therapy Yields Improvements in AADC-Deficiency: Paul Wuh-Liang Hwu, MD, PhD
The professor from National Taiwan University Hospital discussed 3 studies of PTC-AADC for AADC deficiency.
Challenges in Treating Relapsed/ Refractory DL B-Cell Lymphoma
Experts discussed challenges with treating relapsed or refractory diffuse large B-cell lymphoma.
CAR T-Cell Therapy and COVID-19 Vaccinations: Muhammad Bilal Abid, MD, MRCP
The assistant professor of medicine from Medical College of Wisconsin discussed the potential impact of CAR T-cell therapy on the efficacy of COVID-19 vaccinations.
Fabry and Gaucher Gene Therapies Well-Tolerated
AVROBIO shared interim safety data from ongoing phase 1 and 2 trials.
The Future of Frontline CAR T in Mantle Cell Lymphoma: Brad S. Kahl, MD
The professor of medicine at Washington University School of Medicine in St. Louis discussed the potential utility of frontline CAR T-cell therapy in mantle cell lymphoma.
Tofersen Fails to Meet Endpoint in Familial ALS Despite Favorable Trends
Tofersen failed to meet its primary end point in change from baseline in ALSFRS-R but differences in total cerebrospinal fluid SOD1 protein and neurofilament light chain were observed.
Diabetes Cell Therapy Improves Insulin Production in First Patient Dosed
The patient’s daily insulin requirement went from 34 units a day at baseline to 2.9 units a day 90 days after treatment.
Access to Gene Therapies for Rare Diseases: Barry Byrne, MD, PhD
The director of the Powell Gene Therapy Center at the University of Florida discussed improving access to gene therapies for patients with rare diseases worldwide.
CLL Cells Edited With CRISPR and mRNA-Based Editing
More than 70% to 80% of homozygous knockout of a given gene can be achieved by electroporating CLL cells with ribonucleoprotein complexes that harbor recombinant Cas9 nuclease and synthetic guides.
CAR T-Cell Therapy for Non-Hodgkin Lymphoma
The chief of oncology and hematology at University of Nebraska Medical Center discussed the use of CAR T-cell therapy in non-Hodgkin lymphoma.
Solid Tumors Targeted in Next-Generation CAR T Collaboration
The collaboration combines AdAlta’s i-bodies and Carina Biotech’s chemokine receptor platform.
Cilta-Cel Outcomes in R/R Multiple Myeloma: Omar Castaneda Puglianini, MD
The hematology and medical oncologist at Moffitt Cancer Center discussed results from the phase 1b/2 CARTITUDE-1 trial of cita-cel in R/R MM.
