Around the Helix: Cell and Gene Therapy Company Updates – July 6, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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.
Connecting Patient Advocacy and Industry for Gene Therapy Development
Susan Ruediger, founder and chief mission officer, CMT Research Foundation, discussed the panel discussion she participated in at BIO 2022.
Pursuing Slow but Steady Gene Therapy Development
Michael Parini, chief executive officer and director, Freeline Therapeutics, discussed the company’s pipeline and mission.
Sarepta’s Duchenne Muscular Dystrophy Gene Therapy Placed on Clinical Hold
The hold was related to a serious adverse event of hypomagnesemia in an ongoing phase 2 clinical trial.
Late-Onset Pompe Disease Gene Therapy Trial Placed on Clinical Hold
The hold comes after a serious AE of peripheral sensory neuropathy.
David Weinstein, MD, on Delivering Gene Therapy Directly to the Brain
The senior vice president of clinical development at Passage Bio discussed intracisternal magna administration of PBGM01.
Nilanjan Ghosh, MD, PhD, on Real-World Efficacy of CAR T-Cell Therapies
The chief of the lymphoma division and oncologist at Levine Cancer Institute discussed data from real-world experience studies.
Liso-Cel Approved for Second-Line Large B-Cell Lymphoma
The approval builds off of liso-cel's (Breyanzi) previous indication for third-line or later treatment in LBCL.
CGTLive’s Weekly Rewind – June 24, 2022
Review top news and interview highlights from the week ending June 24, 2022.
Val-Rox Approval Favored in Europe, Stalled in US
BioMarin received a positive CHMP opinion after the FDA delayed val-rox's BLA filing for the second time.
Krystal Seeks Approval for Dystrophic Epidermolysis Bullosa Gene Therapy
B-VEC recently showed efficacy in the phase 3 GEM-3 trial.
Steven W. Pipe, MD, on Challenges in Gene Therapy Trials for Hemophilia
The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed common challenges with gene therapy clinical trials.
Huntington Disease Gene Therapy Promotes Reduction in Mutant Huntington Protein, Neurofilament Light
NfL peaked 1-month post-treatment as expected and returned to near baseline at 12 months.
Gene-Mediated Immunotherapy for Ovarian Cancer Clears Safety Hurdle, Will Continue Dosing
The phase 1/2 OVATION 2 study is expected to complete enrollment by the third quarter of this year.
Thomas Martin, MD, on Exciting Cell Therapy Options in Multiple Myeloma
The clinical professor of medicine, Helen Diller Family Comprehensive Cancer Center, UCSF, discussed cilta-cel and upcoming cell therapies.
