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
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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.
Around the Helix: Cell and Gene Therapy Company Updates – July 6, 2022
Stefanie Bailey, PhD, on the Role of Interferon-γ in CAR T-Cell Therapy
The research fellow from Harvard Medical School discussed blocking IFNg as a potential approach to reduce toxicities.
Vertex’s Type 1 Diabetes Islet Cell Replacement Therapy Resumes Dosing
The FDA has lifted a clinical hold on the trial of VX880 placed in May 2022 due to insufficient information.
Adham Bear, MD, On Immunotherapy Targeting Mutant KRAS
The instructor of Medicine, Division of Hematology and Oncology, Hospital of the University of Pennsylvania discussed his thoughts on the potential of targeting mutant KRAS with immunotherapy.
Daniel Judge, MD, on Making Genetic Testing, Insights More Mainstream
MUSC recently launched In Your DNA SC, a 4-year study to collect and glean insights from genetic testing in the general population.
Jakob Dupont, MD, on ATA188’s Potential in All Forms of Multiple Sclerosis
The global head of research and development at Atara Biotherapeutics discussed ATA188 ahead of anticipated data readouts this month.
Mazyar Shadman, MD, MPH, on MB-106's Potential in Follicular Lymphoma
The associate professor at Fred Hutch Cancer Center discussed data presented at the EHA 2022 Congress.
CGTLive’s Weekly Rewind – July 1, 2022
Review top news and interview highlights from the week ending July 1, 2022.
Gene Therapy for Canavan Disease Shows Early Promise in Safety and Pharmacodynamics
A patient showed more than a 50% reduction in N-acetylaspartate (NAA) in brain white matter at 3 months post-treatment.
Christian Hinrichs, MD, on Improving Solid Tumor Targeting With Engineered T-Cell Therapies
The chief of cancer immunotherapy at Rutgers Cancer Institute discussed clinical results with different cell therapy approaches.
Gene Therapy Improves Outcomes, Yields Sustained Aflibercept Expression in Wet AMD
ADVM-022 was also granted PRIME designation by the EMA on June 24, 2022.
Breanna DiAndreth, PhD, on Improving Selective Targeting of Tumor Cells
The scientist at A2 Biotherapeutics discussed the company’s dual-receptor Tmod technology.
Around the Helix: Cell and Gene Therapy Company Updates – June 29, 2022
Peter Bross, MD, on Regulating Cell Therapies: Lessons Learned
The acting chief of the oncology branch of the Center for Biologics Evaluation and Research at the FDA discussed the evolution of cell therapies in the past decade.
Adrenomyeloneuropathy Natural History Study to Inform Gene Therapy Research
It will be the first AMN clinical study to utilize wearable technology for monitoring patient activity and outcomes.
Gene Therapy for Chronic Pain To Be Explored Under NINDS Grant
Benjamin Harrison, BSc, PhD, assistant professor of biochemistry and nutrition at UNE, shared his thoughts on the research.
