SPG50 Gene Therapy Warrants Further Study for Spastic Paraplegia
MELPIDA was well-tolerated in patients and nerve conduction was stable or improved after treatment.
Travis Drow, BS, on Evaluating Engineered Tregs for Potential in Treating Multiple Sclerosis
The research scientist at Seattle Children's Research Institute discussed mouse model research he presented at ASGCT’s 2024 Meeting.
Patient With DMD Dies in Pfizer’s Phase 2 Gene Therapy Trial
In response to the death, Pfizer is pausing dosing associated with the crossover portion of the phase 3 CIFFREO trial.
TDT/SCD Lentiviral Therapy Boasts Quick, Well-Tolerated Engraftment in Patients
KL003 favorably compares to platelet and neutrophil recovery rates of approved gene therapies.
Holly Peay, PhD, on Patient Preference Research as a Means of Patient Expression
The senior research scientist at RTI International discussed how patient preference studies can help patients with rare diseases have their voices heard.
First Patient Set to Receive bluebird’s Approved Sickle Cell Gene Therapy, Lyfgenia
Lovo-cel was approved as Lyfgenia in December 2023.
Carlos A. Ramos, MD, on Alternatives to T-Cell Therapy
The professor of medicine at Baylor College of Medicine discussed research with NK-T cells and alternatives to αβ T-cells.
Intellia’s Phase 3 Clinical Trial for CRISPR-Based Gene Therapy Enrolls First Patient
The first patient in the trial, which is evaluating NTLA-2001 for the treatment of ATTR Amyloidosis, was enrolled at MedStar Washington Hospital Center.
Prime Medicine’s Gene-Edited Stem Cell Product PM359 Cleared for US Trial in Chronic Granulomatous Disease
The company anticipates that initial data from the study will be announced next year.
Advances in CRISPR/Cas9 Gene Editing Will Be Highly Represented in ASGCT Presentations This Year
Terence R. Flotte, MD, the vice president of ASGCT, discussed what attendees can expect at the ASGCT Annual Meeting this year.
Holly Peay, PhD, on Keeping Patient Preference in Focus
The senior research scientist at RTI International discussed the progress that has taken place in recent years with regard to patient preference research.
CGTLive®’s Weekly Rewind – May 3, 2024
Review top news and interview highlights from the week ending May 3, 2024.
Terence R. Flotte, MD, on the Upcoming ASGCT Annual Meeting and Recent Trends in Gene Therapy
The vice president of ASGCT discussed important research that will be represented at the upcoming conference and challenges for the field to address in the future.
ODAC Supports MRD Endpoint in Accelerated Approval for Multiple Myeloma
The ODAC unanimously voted in favor of the use of minimal residual disease negativity.
Labcorp’s Companion Diagnostic Assay for Beqvez Receives FDA Approval
Testing for the presence of neutralizing antibodies is an essential step before patients can receive Beqvez.
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.
