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.
Around the Helix: Cell and Gene Therapy Company Updates – November 29, 2023
Autolus Submits Obe-Cel BLA for Adult R/R B-ALL
The company also plans to submit an MAA to the EMA in the first half of 2024.
Eileen Tzng, BA, on Exploring Exosome and RNA-based Treatment Methods for Heart Failure
The life science research professional at Stanford University discussed preclinical research she presented at the American Heart Association’s Scientific Sessions 2023.
FDA to Investigate Risk of T-Cell Malignancy After CAR T-Cell Therapy
The FDA has received reports of multiple T-cell malignancies in patients after approved CAR-T treatment.
Second Gene Therapy for Epidermolysis Bullosa up for Priority Review
Pz-cel has a PDUFA date of May 25, 2024.
Nexcella’s Light Chain Amyloidosis CAR-T NXC-201 Expands Clinical Trial Activities Into the US
The CAR-T was already being assessed in the ongoing phase 1a/1b NEXICART-1 clinical trial in Israel ahead of this IND clearance by the FDA.
Gracell’s CAR T-Cell Therapy to be Evaluated in Lupus
GC012F recently yielded a 100% ORR in an investigator-initiated trial in patients with newly diagnosed multiple myeloma.
Krystal’s Dystrophic Epidermolysis Bullosa Gene Therapy B-VEC Under Review in EU
CHMP is expected to give an opinion on the MAA in the second half of next year.
Alfonso Sabater, MD, PhD, on Bringing Gene Therapy to Ophthalmology
The associate professor of clinical ophthalmology at University of Miami discussed potential future areas of investigation for gene therapy in ocular diseases.
First Patient Dosed With KH631 Gene Therapy in Phase 1 Trial of Wet AMD
The phase 1 VAN-2201 clinical trial (NCT05657301) is assessing the AAV-delivered gene therapy from Chengdu Origen Biotechnology and Vanotech in 5 dose cohorts of 25 total patients.
Novel Vector Development is Essential as Gene Therapy Emerges as a New Therapeutic Modality in Cardiology
Roger Hajjar, MD, the director of the Mass General Brigham Gene and Cell Therapy Institute, spoke about his presentation on gene therapy for cardiologists at AHA’s 2023 Scientific Sessions.
