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.
James Beck, PhD, on Identifying Genetic Variants of Parkinson Disease
The chief scientific officer and senior vice president of the Parkinson’s Foundation discussed mutations the Parkinson’s Foundation has identified.
Around the Helix: Cell and Gene Therapy Company Updates – July 13, 2022
First Patient With Familial Hypercholesteremia Dosed With Base-Editing Therapy
Verve Therapeutics is currently conducting a clinical trial in New Zealand and expects to be able to open trials in the UK and US later in 2022.
Susan Ruediger on Pursuing Gene Therapies for CMT
Susan Ruediger, founder and chief mission officer, CMT Research Foundation, discussed the foundation’s mission.
TCR T Cell Therapy for HBV-Related Liver Cancer Cleared for US Trial
SCG101 will be evaluated in trials across China, Singapore, and the US.
Barbara Lavery on Collaborating to Develop Cancer Therapies
The chief program officer of the Alliance for Cancer Gene Therapy discussed the work the nonprofit is doing.
Freeline Moves Closer to Identifying Hemophilia B Gene Therapy Dose
A second cohort has been dosed with 7.7e11 vg/kg to hopefully confirm the dose for phase 3 trials.
CAR Macrophage Therapy Continues to be Well-Tolerated in Solid Tumors
A phase 1 trial has completed enrollment in its first cohort and is now enrolling the second.
The Growing Popularity of Gene Therapy for Retinal Diseases
Foundation Fighting Blindness is helping to support the development of gene therapies for retinal diseases.
Gavin Macbeath, PhD, on Screening for Off-Target Effects
The chief scientific officer at TScan Therapeutics discussed the company’s SafetyScan technology.
Legend Biotech Drops Lymphoma CAR T-Cell Therapy
Legend Biotech seeks to prioritize other product candidates in its pipeline.
Canavan Disease Gene Therapy Continues to Show Promising Data
Myrtelle’s rAAV-Olig001-ASPA was granted fast track, rare pediatric disease, and orphan drug designations in March 2022.
Serena De Vita, MD, PhD, on Speedy Manufacturing of PHE885 for Multiple Myeloma
The senior director and clinical program leader, Translational Clinical Oncology at Novartis Institutes for BioMedical Research, discussed new data from the phase 1 study.
Jeffrey Miller, MD, on Improving NK Cells for Solid and Hematologic Malignancies
The professor of medicine and University of Minnesota Medical School discussed the advantages and unmet needs of natural killer cells in oncology.
