Sarepta, Broad Institute Strike Licensing Agreement for New Class of AAV Vectors for Gene Therapy
MyoAAV was shown to deliver 25 to 50 times greater gene expression in multiple skeletal muscles compared with natural AAV serotypes.
Multiple Myeloma CAR T-Cell Therapy Promotes Durable Responses
CT103A, a fully human BCMA-directed CAR T-cell therapy, demonstrated deepening efficacy with an acceptable toxicity profile, according to updated data from the phase 1/2 FUMANBA-1 trial.
UniQure Pauses High Dose of AMT-130 Huntington Disease Gene Therapy for Severe Adverse Events
No such adverse events have occurred in the lower-dose group.
Nilanjan Ghosh, MD, PhD, on Paving the Way for CAR T Therapies in Lymphomas
The chief of the lymphoma division and oncologist at Levine Cancer Institute discussed work that needs to be done to support the use of CAR T therapy.
The Power of Partnership in CMT Gene Therapy Research
Susan Ruediger, founder and chief mission officer, CMT Research Foundation, discussed the origins of the CMTRF.
CGTLive’s Weekly Rewind – August 5, 2022
Review top news and interview highlights from the week ending August 5, 2022.
Umbilical Cord Lining Stem Cell Therapy Trial Initiates for Autoimmune Disorders
The trial is being launched by the University of Florida Center for Regenerative Medicine in collaboration with RESTEM.
Allogeneic Cell Therapy Trial to Initiate in Ovarian Cancer
Avenge Bio previously presented positive preclinical data at the ASGCT 2022 meeting.
Craig M. McDonald, MD, on Safety and Efficacy of DMD Cell Therapy
The principal investigator of the HOPE-2 trial discussed safety and efficacy of Capricor’s CAP-1002.
Around the Helix: Cell and Gene Therapy Company Updates – August 3, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
REGENXBIO's MPSII Gene Therapy Charts Course to Approval
The FDA will consider RGX-121's BLA in an accelerated approval pathway.
Diabetic Kidney Disease Cell Therapy Trial Completes Enrollment
Topline results are expected in the first quarter of 2023.
Richard Fessler, MD, on Progress With Cell Therapy in Spinal Cord Injury
The professor of neurosurgery at Rush University Medical School discussed the potential of Lineage Cell’s LCTOPC1 cell therapy.
IND Cleared for Dual-Targeted CAR T Therapy for B-Cell Malignancies
UCART2022 is an allogeneic therapy that targets both CD20 and CD22.
FDA Accepts IND Application for Cystic Fibrosis Gene Therapy KB407
The phase 1 clinical trial from Krystal Biotech is expected to begin this year.
HIV Gene Therapies Continue to Move Through the Pipeline With Promise
Seven participants have been dosed with AGT103-T, an autologous T-cell therapy currently being evaluated in a phase 1 trial.
COVID-19 Cell Therapy Trial Dosing Complete With Good Safety Profile
Elderly or high-risk patients with COVID-19 received 1 of 4 escalating dose levels.
Clinical Hold Lifted on Celyad's Phase 1b Trial in Refractory Metastatic Colorectal Cancer
The company voluntarily paused the trial in February 2022 to investigate 2 reported deaths in the trial.
James Beck, PhD, on Laying Groundwork for Gene Therapy in Parkinson Disease
The chief scientific officer and senior vice president of the Parkinson’s Foundation discussed remaining research and education needs to support the future of genetic medicines.
Gene Therapy: The Future of Rare Disease Treatment
Experts shared opinions about the future of gene therapy in sickle cell and other rare diseases.
CGTLive’s Weekly Rewind – July 29, 2022
Review top news and interview highlights from the week ending July 29, 2022.
Sarepta Planning BLA Submission of DMD Gene Therapy
The announcement follows positive data updates from the ENDEAVOR and SRP-9001-101 studies.
Mazyar Shadman, MD, MPH, on Addressing Unmet Needs in Follicular Lymphoma With MB-106
The associate professor at Fred Hutch Cancer Center discussed next steps with the clinical trial being conducted Fred Hutch Cancer Center.
Natural Killer Cell Therapy Trial Doses First Patient With NSCLC
CYTO-102 is being developed by City of Hope and CytoImmune.
Editas' Gene Therapy Trial for Sickle Cell Initiates After Hold Lifted
The company announced successful neutrophil and platelet engraftment for the first patient dosed.
Nilanjan Ghosh, MD, PhD, on New Avenues of CAR T Research in Lymphoma
The chief of the lymphoma division and oncologist at Levine Cancer Institute discussed new CAR T-cell therapies being investigated for hematologic malignancies.
Gastric/Gastroesophageal Junction Cell Therapy Trial Doses First Patient
CYNK-101 has previously been granted fast track and orphan drug designation.
Around the Helix: Cell and Gene Therapy Company Updates – July 27, 2022
First Patient Dosed in RAG1-SCID Gene Therapy Trial
Mustang Bio plans to add more clinical trial sites in the near future.
Liso-Cel Approval Expands CAR T-Cell Access in LBCL
Nilanjan Ghosh, MD, PhD, discussed the impact of liso-cel's approval.
