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
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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 Hearth 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.
Fernanda Mesquita, PhD, on Using LAMA2-Exosomes to Improve Cardiomyocyte Survival in Stroke
The research associate at the The Texas Heart Institute discussed preclinical research she presented at AHA’s 2023 Scientific Sessions on MSC-derived exosomes.
Sharon Hesterlee, PhD, on Collaborating to Expand Therapy Development to More Dystrophies
The chief research officer of the Muscular Dystrophy Association discussed the need for disease-modifying therapies in other muscular dystrophies and programs the association has announced to this end.
Enabling Wider Antigen Selection With Logic-Gated CAR T-Cell Therapy for Solid Tumors
Maria Pia Morelli, MD, PhD, assistant professor, MD Anderson Cancer Center, discussed her work on the EVEREST-1 trial of logic-gated CAR T-cell therapy currently being evaluated in colorectal and pancreatic cancers.
CGTLive’s Weekly Rewind – November 24, 2023
Review top news and interview highlights from the week ending November 24, 2023, including coverage from AHA 2023.
BioCardia Gets FDA Greenlight for Modified CardiAMP Heart Failure II Trial
The CardiAMP autologous cell therapy is being assessed for ischemic heart failure, with a new phase 3 trial that includes modified eligibility requirements and end points.
More Data Supports Approval as Lifileucel Review for Melanoma Pushed Back
The new PDUFA date of lifileucel is set for Februrary 24, 2024.
Around the Helix: Cell and Gene Therapy Company Updates – November 22, 2023
Thomas Crawford, MD, on Helping Patients With SMA Left Behind by Gene Therapy
The codirector of the MDA Clinic and professor of neurology at Johns Hopkins discussed the importance of continuing to fight for progress with older patients with SMA.
NKGen’s NK Cell Therapy SNK01 Improves Cognitive Function in Alzheimer Disease For up to 11 Weeks
Among 10 patients evaluated compared to their baseline at 1 week after receiving their final dose in the trial, 30% of patients demonstrated clinical improvement on the ADCOMS.
BrainStorm to Meet With FDA on Confirmatory Trial for NurOwn ALS Therapy
After a negative AdComm meeting and withdrawing its BLA, the company seeks guidance on a phase 3 trial.
Xaviar Michael Jones, MD, on Expanding Horizons in RNA Therapeutics Through Collaboration
The postdoctoral research fellow at Cedars Sinai Medical Center discussed the future of RNA therapy in the context of research in systemic sclerosis he presented at AHA’s Scientific Sessions 2023.
BMS and 2seventy’s PDUFA for Abecma in Earlier-Line Multiple Myeloma Pushed to Accommodate New Adcomm Meeting
The date for the AdComm and the new PDUFA date are yet to be announced.
Iovance’s NSCLC Trials Push Second TIL Therapy Closer to Submission
The PDUFA date for Iovance’s lead TIL lifileucel for melanoma was recently pushed back to February 24, 2024.
J. Andrew Livingston, MD, on Starting the Shift Toward Targeted Therapies in Sarcoma
The associate professor at MD Anderson Cancer Center discussed his future outlook and goals for the field of sarcoma.
Safety Monitoring Critical as Cardiology Wades Into Gene Therapy for Hypertrophic Cardiomyopathy
Milind Desai, MD, MBA, the director of the Hypertrophic Cardiomyopathy Center and the vice chair of the Heart Vascular Thoracic Institute at the Cleveland Clinic, discussed the design of Tenaya Therapeutics’ clinical trial evaluating TN-201.
Lepodisiran Reduces Lipoprotein(a) Levels Safely in Phase 1 Data, Paving Way for Further Study
Eli Lilly’s small interfering RNA therapy showed significant serum reductions with a single dose while remaining well-tolerated. A larger phase 2 study is currently ongoing.
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