MAGE-A4 T-Cell Engager Cleared for Phase 1 Trial in Solid Tumors
CDR-Life also recently presented positive preclinical data at the November 2023 SITC Annual Meeting.
M. Peter Marinkovich, MD, on Increased Accessibility With Topical B-VEC DEB Gene Therapy
The associate professor of dermatology at Stanford University discussed the potential impact of Vyjuvek’s approval in the derm field.
Sickle Cell Disease Gene Therapies to Be Part of Initial Focus for Biden-Harris Administration’s CGT Access Model
The new Cell and Gene Therapy Access Model will be led by the Centers for Medicare & Medicaid Services’ Innovation Center.
The Field of Neuromuscular Disease is Entering the Era of Treatment
Barry J Byrne, MD, PhD, the chief medical advisor of MDA, discussed MDA Care Centers and his excitement for the organization’s the Upcoming Clinical & Scientific Conference.
Akouos Seeks to Tackle Otoferlin Gene-Mediated Hearing Loss With Phase 1/2 Clinical Trial and Associated Natural History Study
AK-OTOF delivers transgenes encoding OTOF, the disease-targeted gene, to the inner hair cells of the cochlea via a single unilateral intracochlear administration.
FDA Activity Recap: January 2024 Features Major Approval, New Black Box Warnings, and Updated Guidance Documents
Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive™ team.
Top News in Oncology Cell Therapy for World Cancer Day 2024
For World Cancer Day, held annually on February 4, take a look at the stories that have stood out in oncology cell therapy so far this year.
Epilepsy Cell Therapy NRTX-1001 May Eventually Be Evaluated for Alzheimer Disease
Cory R. Nicholas, PhD, the cofounder and CEO of Neurona Therapeutics, discussed future plans for the expanded applications of the allogeneic regenerative neural cell therapy.
FDA Accepts Adamptimmune’s BLA for Synovial Sarcoma TCR T-cell Therapy Afami-cel With Priority Review
The Prescription Drug User Fee Act (PDUFA) target action date has been set for August 4, 2024.
CGTLive’s Weekly Rewind – February 2, 2024
Review top news and interview highlights from the week ending February 2, 2024.
Julie Kanter, MD, on Developing Recommendations for SCD Post Gene Therapy
The director of the Adult Sickle Cell Clinic and associate professor at University of Alabama Birmingham discussed work the National Alliance of Sickle Cell Centers is conducting.
Ultragenyx Completes Dosing of UX701 Gene Therapy in Patients With Wilson Disease
The ongoing, multistage phase 1/2/3 Cyprus2+ study (NCT04884815) expects to read out initial safety and efficacy data in the first half of 2024.
First Patient With AML Receives CD33-CAR T Cells From Previous HCT Donor
Vor Bio recently also announced data from the phase 1/2 trial of trem-cel.
Marcela Maus, MD, PhD, on New Questions With CAR-T's Rise in Solid Tumors
The Associate Professor of Medicine at Harvard Medical School discussed CAR T’s journey to treating solid tumors.
Neurona Therapeutics NRTX-1001 Has Shown Promising Early Signs of Safety and Efficacy in Drug-Resistant Epilepsy
Cory R. Nicholas, PhD, the cofounder and CEO of Neurona Therapeutics, discussed the ongoing first-in-human clinical trial evaluating the cell therapy for drug-resistant mesial temporal lobe epilepsy.
Alfonso Sabater, MD, PhD, on Further Research With Gene Therapy in Eye Care
The associate professor of clinical ophthalmology at University of Miami discussed the first topical application of gene therapy to the eye.
Around the Helix: Cell and Gene Therapy Company Updates – January 31, 2024
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
FDA Approves Kite’s Axi-Cel Manufacturing Change to Reduce Turnaround Time
Kyverna has also announced data on its proprietary CAR-T manufacturing process, Ingenui-T.
Ahead of Announcing Positive Results Seen in Trial for Gene Therapy FBX-101, Forge Biologics’ CEO Makes Comment Supporting Krabbe Disease’s Addition to RUSP
Efficacy and safety results from the RESKUE clinical trial will be presented at at the WORLDSymposium 2024 on February 9.
Indapta Treats First Patients With Multiple Myeloma, NHL, With NK Cell Therapy
The company also shared that it has manufactured sufficient IDP-023 to supply the Phase 1 clinical trial through the second half of 2024.
Tom E. Howard, MD, PhD, on a Gene Editing Approach to Treating Hemophilia A
The clinical professor in the Department of Human Genetics at University of Texas Rio Grande Valley discussed how a personalized gene editing approach may help patients avoid development of FVIII inhibitors.
Fate and Ono Initiate Enrollment for HER2-CAR T Trial
FT825 is an iPSC-derived multiplexed-engineered, CAR T-cell therapy targeting HER2 with a novel antigen binding domain.
Shankar Musunuri, PhD, on Continuing Research With Gene Therapy in Inherited Retinal Diseases
The chairman, chief executive officer, and cofounder of Ocugen discussed programs including OCU400 and OCU410.
Sarepta Therapeutics' SRP-5051 Demonstrates Ability to Increase Dystrophin Expression in Patients With Duchenne Muscular Dystrophy
SRP-5051 is an investigational peptide-conjugated phosphorodiamidate morpholino oligomer.
Pat Furlong, BSN, RN, on Continuing Research With Muscular Dystrophy After Gene Therapy Approval
The founding president and chief executive officer of Parent Project Muscular Dystrophy discussed the organization’s research imperatives of 2024.
Sarepta Therapeutics' Phase 3 Study for Limb-Girdle Muscular Dystrophy Gene Therapy SRP-9003 Begins Screening Activities
The multinational, open-label EMERGENE study will seek to enroll 15 patients with LGMD2E/R4 in total.
Barry J Byrne, MD, PhD, on the Potential of Directed Evolution to Improve AAV Gene Therapy
The chief medical advisor of the Muscular Dystrophy Association discussed how directed evolution may help develop AAV capsids better suited for treating neuromuscular diseases.
Allogeneic CD22 CAR Cleared for First-in-Human Trial in B-Cell Malignancies
Sana also recently announced data from the first patient treated with its CD19-targeted CAR.
Cell Therapy May Help to Address Unmet Needs in Epilepsy
Cory R. Nicholas, PhD, the cofounder and CEO of Neurona Therapeutics, discussed NRTX-1001, the company’s allogeneic regenerative neural cell therapy currently being evaluated in MTLE.
Sensorion’s OTOF Hearing Loss Gene Therapy Cleared for Phase 1/2 Trial in France
The trial will recruit patients aged 6 to 31 months with the intent of treating them while auditory system plasticity is optimal.
