NIH Strategizes to Streamline Gene Therapy Development
PJ Brooks, PhD, deputy director, Division of Rare Diseases Research Innovation, NCATS, NIH, discussed ongoing initiatives.
Parkinson Gene Therapy Shows Stability, Some Improvements in ON/OFF Time Disease Measures
Based on these data, the phase 2 Regenerate PD trial of AB-1005 will begin enrolling later this year.
FDA Activity Recap: April 2024 Features New Product Approval, 2 Expanded Indication Approvals, and More
Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.
Around the Helix: Cell and Gene Therapy Company Updates – May 1, 2024
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Looking Forward to A Potential First Cell Therapy Approval for Sarcomas
John A. Charlson, MD, associate professor of medicine, Medical College of Wisconsin, discussed advantages of afami-cel for synovial sarcoma and other solid tumors.
ASO Fast Tracked for Myotonic Dystrophy
PGN-EDODM1 is being evaluated in the US and Canada in a phase 1 trial.
ASGCT’s 2024 Annual Meeting Will Showcase the Field’s Rapid Advances
Paula Cannon, PhD, the president elect of ASGCT, discussed things to look forward to at the upcoming Meeting that will be held from May 7 to 11, in Baltimore, MD.
NKGen’s NK Cell Therapy SNK01 Cleared for US Clinical Trial in Parkinson Disease
NKGen anticipates that it will dose the first patient in the study in the second half of this year.
Deepak L. Bhatt, MD, MPH, MBA, on Adopting Novel Therapies into Cardiovascular Medicine
The director of the Mount Sinai Fuster Heart Hospital discussed trends in research and challenges with adopting new therapies.
TMZ γδ T-Cell Regimen May Have Potential in Treating Glioblastoma
A phase 1 trial evaluating INB-200 has shown a PFS benefit in treated participants.
Fungal Infections May Pose a Greater Risk for Future CAR-T Applications
Jessica S. Little, MD, a transplant infectious diseases physician at Dana-Farber Cancer Institute, discussed research on fungal infections in patients receiving HSCT and CAR-T.
Ferring Testing Bladder Cancer Gene Therapy Adstiladrin’s Mettle in Real-World Setting With ABLE-41 Observational Study
In anticipation of Bladder Cancer Awareness Month, CGTLive® has decided to take a closer look at this noninterventional real-world study.
Taysha Transfers Deprioritized Gene Therapy Programs Back to Partners
The company’s TSHA-201 AAV gene therapy for Rett syndrome is its current priority.
Jeffrey Chamberlain, PhD, on Bringing Back the Focus to Basic Research for ASGCT 2024
The McCaw Endowed Chair of Muscular Dystrophy at University of Washington gave a background on ASGCT, its founding, and how far the field has come since.
Amit Soni, MD, on Challenges to the Uptake of Gene Therapy for Hemophilia
The medical director of the Center for Inherited Blood Disorders discussed barriers to the widespread adoption of val-rox by the hemophilia A community.
Taking Lessons Learned With CAR-T in Oncology to the Autoimmune Space
David Porter, MD, director of Cell Therapy and Transplant, Penn Medicine discussed how experience in oncology enabled the logical expansion.
First Patient With Diabetic Foot Ulcers Screened for SkinTE Pivotal Trial
Another recent program in the investigational landscape was Helixmith’s deprioritized Engensis diabetic neuropathy gene therapy.
Trial for Regeneration Biomedical’s Alzheimer Disease Stem Cell Therapy Doses First Patient
The first-in-human, open-label, single-arm trial is expected to enroll 9 participants over the course of a year.
Jonathan W. Weinsaft, MD, Future Research With Gene Therapy for Cardiovascular Disease
The chief of cardiology at Weill Cornell Medical College shared his outlook on the future of gene therapy research in the field.
The CAR-T Versus Bispecific Antibody Debate in Third Line LBCL
Emilie Aschenbrenner, PharmD, BCOP, a hematology coordinator for pharmacy at Froedtert and the Medical College of Wisconsin, discussed the advantages of each of the 2 modalities in the third and second line settings.
CGTLive®’s Weekly Rewind – April 26, 2024
Review top news and interview highlights from the week ending April 26, 2024.
FDA Approves Fidanacogene Elaparvovec-dzkt for Moderate to Severe Hemophilia B
The decision was supported by the phase 3 BENEGENE-2 trial, which suggested that the Pfizer gene therapy was superior to standard of care FIX prophylaxis.
iNKT Cells Safe, Shows Anti-Inflammatory Response in Patients With SARS-CoV-2
The cells may have potential to modulate disease in other cases of respiratory disease and critical illness.
Amit Soni, MD, on Treating the First Patient With Hemophilia A Gene Therapy in the Real-World Setting
The medical director of the Center for Inherited Blood Disorders discussed the entrance of BioMarin’s val-rox into his center’s practice.
Artiva Biotherapeutics Doses First Patient With NK Cell Therapy AlloNK in Lupus Trial
AlloNK is being administered in the trial alone or alongside 1 of 2 antiCD20 monoclonal antibodies: rituximab or obinutuzumab.
Omid Hamid, MD, on Post-Approval Research With Lifileucel
The professor from Cedars Sinai discussed data he is anticipating from further investigations into lifileucel.
Lineage Cell to Evaluate Novel Delivery of OPC1 Cell Therapy for Spinal Cord Injury
The company expects to open the first clinical site in the DOSED study in the second quarter of 2024.
Paula Cannon, PhD, on What Attendees Can Look Forward to at ASGCT’s 2024 Meeting
The President Elect of ASGCT and a distinguished professor of microbiology at Keck School of Medicine of USC also discussed recent milestones in gene therapy.
Around the Helix: Cell and Gene Therapy Company Updates – April 24, 2024
First Patient With Gastric Cancer Dosed in iNKT/CTLA-4 Inhibitor/Anti-PD-L1 Trial
A case study previously described successful immune modulation with AgenT-797 in a patient with high-risk gastric cancer leading to a partial response.
