ProQR Pivots From Ophthalmic to CNS, Liver RNA Therapies
The move comes after the EMA recommended an additional clinical trial of sepofarsen before applying for marketing authorization.
Merit Cudkowicz, MD, on Evaluating Mesenchymal Cell Therapy for ALS
BrainStorm Cell Therapeutics has decided to submit a BLA for NurOwn in ALS following an erratum to a phase 3 trial.
Genprex to Evaluate Higher Dose of REQORSA in NSCLC
A safety review committee approved the dose-escalation after reviewing the first cohort of patients.
Pompe Disease mRNA Therapy Receives FDA Orphan Drug Designation
Preclinical data showed a reduction of Gys1 mRNA and protein of more than 80% in multiple skeletal tissues.
Bristol Myers to Fund Inflammatory Bowel Disease Treg Therapies
GentiBio will use their proprietary regulatory T cell platform to develop the engineered therapies against multiple targets.
Shannon L. Maude, MD, PhD, on Favorable Safety of Tisagenlecleucel in Pediatric B-ALL
The oncologist from Children's Hospital of Philadelphia discussed long-term safety data with tisagenlecleucel in pediatric patients with acute lymphoblastic leukemia.
CGTLive’s Weekly Rewind – August 12, 2022
Review top news and interview highlights from the week ending August 12, 2022.
Zolgensma Gene Therapy Linked to 2 Deaths in SMA Patients, Novartis Reports
The 2 deaths, due to acute liver failure, occurred in patients treated in Kazakhstan and Russia.
ALS Cell Therapy Receives US Patent
AstroRx previously demonstrated clinically meaningful reductions in the rate of disease progression.
Frontotemporal Dementia Gene Therapy Trial Initiates Dosing
PBFT02, developed by Passage Bio, is delivered via intra cisterna magna administration to the brain.
CRISPR Therapeutic Ready to Dose in Duchenne Muscular Dystrophy
A single patient will be dosed with CRD-THM-001 in an upcoming trial.
NK Cell Therapy Trial Doses First Patient With Lymphoma
The FDA recently accepted the company’s BLA for omidubicel for priority review.
Around the Helix: Cell and Gene Therapy Company Updates – August 10, 2022
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Ide-Cel Shows Benefit in Earlier Use in R/R Multiple Myeloma
A recent comparison of health-related quality of life scores also favored ide-cel over belantamab mafodotin treatment.
IND Cleared for Non-Hodgkin Lymphoma T-Cell Therapy
Marker will initiate a company-sponsored study after an initial trial at Baylor College of Medicine showed positive safety data.
Monitoring and Support for Patients Receiving CAR T-Cell Therapy
Experts discuss providing supportive care to patients receiving CAR T-cell therapy.
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
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.
