Lovo-Cel Efficacy Maintained at 60 Months for Preventing VOEs in SCD
Long-term data on lovo-cel gene therapy for sickle cell disease (SCD) reveals sustained efficacy at 60 months.
Beti-cel Displays Long-Term Success in Adult and Pediatric Transfusion-Dependent β-Thalassemia
Bluebird bio’s gene therapy, marketed as Zynteglo, showed successful rates of transfusion independence up to 9 years with a reasonable safety profile in data presented at ASH 2023.
Hemophilia B Gene Therapy Etranacogene Dezaparvovec Shown to Be Safe and Effective in Subset of Clinical Trial Patients With Comorbid HIV
There were no treatment-related serious adverse events reported.
FDA Experts Weigh In on Exa-cel and Lovo-cel Approvals for Sickle Cell and Corresponding Black Box Safety Warnings
The FDA’s Nicole Verdun, MD; and Peter Marks, MD, PhD, offered insight on the recent approvals of exa-cel and lovo-cel, answering questions about the safety of the gene therapies and the continued collection of data on their use.
CGTLive’s Weekly Rewind – December 8, 2023
Review top news and interview highlights from the week ending December 8, 2023.
FDA Approves bluebird bio's Lovo-Cel Gene Therapy for Sickle Cell Disease
The decision was supported by efficacy data from 36 patients from the ongoing phase 1/2 HGB-206 trial (NCT02140554) and 2 patients in the phase 3 HGB-210 trial (NCT04293185).
FDA Approves Exa-cel, Vertex and CRISPR Therapeutics’ Gene Therapy, for Sickle Cell Disease
The FDA’s decision was based on data from the CLIMB-121 and CLIMB-131 clinical trials, making it the first CRISPR-based gene therapy to be approved in the US.
Sukumar Nagendran, MD, on Addressing Unmet Needs in Rett Syndrome With Gene Therapy
The president and head of R&D at Taysha Gene Therapies discussed the early clinical evaluation of TSHA-102.
Benitec Biopharma’s Oculopharyngeal Muscular Dystrophy Gene Therapy Trial Doses First Patient
Benetic anticipates an announcement of interim results sometime around the midpoint of 2024.
M. Peter Marinkovich, MD, on Developing B-VEC, First Approved Derm Gene Therapy
The associate professor of dermatology at Stanford University discussed his experiences investigating and helping to develop Vyjuvek, approved for treating DEB.
MSC Therapy Improves Pain, Stiffness, Function in Osteoarthritis Compared to Placebo
Stempeutics mesenchymal stem cell therapy is approved in India for treating critical limb ischemia due to multiple causes.
Around the Helix: Cell and Gene Therapy Company Updates – December 6, 2023
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Adaptimmune Submits Afami-Cel First Engineered TCR-T Therapy BLA for Synovial Sarcoma
The cell therapy yielded a median overall survival of around 17 months compared with a historical OS of less than 12 months.
David Porter, MD, on the Importance of Collaboration for Investigating Cell Therapy in Autoimmune Disease
The director of cell therapy and transplant at Penn Medicine discussed the seminar he gave at the inaugural Cell Therapy for Autoimmune Disease Summit.
Tarlatamab Bispecific T-Cell Engager Yields Responses in Small Cell Lung Cancer
ORR was 40% in the 10-mg group and 32% in the 100-mg group.
Maria Pia Morelli, MD, PhD, on Investigating TROP2-Targeting CAR-NK Therapy in GI, Breast, Ovarian Cancers
The assistant professor at MD Anderson Cancer Center discussed ongoing trials at MD Anderson and the TROP2 target for cell therapy.
After Elevidys: DMD Advocacy Past First Gene Therapy Approval
For This Year in Medicine, CGTLive looks back at a landmark first approval, the new treatment opportunities it has provided, and the unmet needs in the field it has brought to light.
Direct Biologics’ Phase 1 Clinical Trial Seeks to Treat Crohn Disease With Extracellular Vesicles
In observance of Crohn and Colitis Awareness Week, observed from December 1 to 7, CGTLive™ took a closer look at the clinical evaluation of the MSC-derived therapy, ExoFlo.
Julie Kanter, MD, on Preparing for Anticipated Approval of Lovo-Cel for Sickle Cell
The director of the Adult Sickle Cell Clinic and associate professor at University of Alabama Birmingham discussed the anticipated approval of the first gene therapy for SCD.
Patient Eligibility Expanded for Phase 1/2 REVEAL Trial of TSHA-102 in Rett Syndrome
Health Canada has greenlit a protocol amendment for Taysha Gene Therapies’ trial to include patients aged 12 years and older, rather than only adult patients.
Mark Walters, MD, on Issues Facing First Possible Sickle Cell Gene Therapy Approval
The professor in residence of pediatrics at University of California San Francisco discussed factors that will limit the potential first approval, including access and policy.
First Patient With Hemophilia A Dosed in New Gene Therapy Trial
ASC618 is designed to minimize cellular stress and may increase durability of gene therapy for hemophilia.
AMT-260 miRNA Gene Therapy Displays Preclinical Promise Ahead of First-in-Human Trial
uniQure’s AAV9-vector therapy carrying 2 small interfering RNAs targeting GRIK2 was well-tolerated, with a hopeful risk-benefit ratio. A phase 1/2 is set to begin recruitment in late 2023.
Ula V. Jurkunas, MD: CALEC Transplant Feasible for Limbal Stem Cell Deficiency
The associate professor of ophthalmology at Harvard Medical School discussed early results from a phase 1/2 trial from Mass Eye and Ear and partners.
CGTLive’s Weekly Rewind – December 1, 2023
Review top news and interview highlights from the week ending December 1, 2023.
Carisma to Investigate New CAR Monocyte Therapy in Solid Tumors
Meanwhile, the company’s lead CAR Macrophage candidate CT-0508, continues to show efficacy in multiple solid tumor trials.
Tom Van Blarcom, PhD, on Bringing CAR-T Constructs From Oncology to B-Cell Driven Autoimmune Disease
The senior vice president and head of research at Kyverna Therapeutics discussed the company’s investigational CAR-T therapies, KYV-101 and KYV-201.
uniQure Expanding Clinical Pipeline to Fabry Disease
The IND clearance sets AMT-191 to be evaluated in a clinical trial alongside the company's gene therapies for ALS and Huntington disease.
Marcela Maus, MD, PhD, on New Approaches to CAR T-Cell Therapy for Solid Tumors
The Associate Professor of Medicine at Harvard Medical School discussed upcoming research and strategies to combat present hurdles in the treatment field.
Tiffany Chen, PhD, on the Role of Preclinical Models in Cell Therapy Research for Autoimmune Disease
The vice president of discovery at GentiBio discussed the panel she participated in at the inaugural Cell Therapy for Autoimmune Disease Summit.
