OTOF Gene Therapy Restores Hearing in Children With Autosomal Recessive Deafness 9
Speech perception was also greatly improved in the children with hearing recovery.
First Patient With mCR Prostate Cancer Receives NK-Engaging INKmune
The immune therapy is a replication-incompetent human tumor cell line designed to conjugate to and prime resting natural killer cells.
CARsgen Hopes to Show Continued GI Cancer Control in ELIMYN18.2 Trial
The trial is assessing satri-cel CAR T-cell therapy, which is currently on clinical hold due to CMC concerns.
No AEs 23 Days Out for First Patient With NSCLC Receiving Deltacel
Kiromic announced that the first patient had been dosed with the gamma delta cell therapy in December 2023.
Satri-Cel Shows Some Disease Control in Gastric/Gastroesophageal and Pancreatic Cancer
Dose level 3 showed the most efficacy and has been selected for the phase 2 portion of the trial.
REGENXBIO’s Wet AMD Gene Therapy Continues to Show Stable BCVA, Reduced Treatment Burden
Updated data from the AAVIATE trial were presented at the Hawaiian Eye and Retina meeting.
CARsgen Gets IND Clearance for GPC3-Targeted CAR for Hepatocellular Carcinoma
Three of CARsgen’s CAR therapies are currently on clinical hold due to CMC questions.
Vertex Nets Early Casgevy Approval for Transfusion-Dependent Thalassemia
The therapy’s original PDUFA date was March 30, 2024, but the TDT approval followed only a month after exa-cel's sickle cell approval.
MultiStem Cell Therapy Not Superior to Placebo in Patients With Ischemic Stroke
Further research is needed to determine if cell therapy may be beneficial to certain subgroups of patients.
Cell Therapy Not Superior to Corticosteroid Injections for Knee Osteoarthritis
The authors noted that this kind of trial is exceedingly complex and future papers will propose further conclusions.
Neurona's Interneuron Cell Therapy Looks Promising in Drug-Resistant Mesial Temporal Lobe Epilepsy
The trial is recruiting 5 more participants for the highest dose cohort following a positive DSMB review.
Kadimastem and iTolerance Work to Bring Immunosuppression-Free Diabetes Cell Therapy to Trials
Kadimasem’s lead clinical candidate, AstroRx, was recently cleared for trials in the US for people with ALS.
Atara’s Cell Therapies Rise for EBV+ PTLD, Sink for Multiple Sclerosis After Data Updates
Atara plans to submit a BLA for tab-cel, approved in Europe, in the second quarter of 2024.
Ornithine Transcarbamylase Deficiency Gene Therapy Trial Begins Recruitment
University College London has initiated the single site trial at Great Ormond Street Hospital, London.
Cystic Fibrosis Gene Therapy Well-Tolerated, Yields Transgene Expression in All 7 Participants
There was 1 serious AE of pneumonia which was found to be possibly related to 4D-710.
CRISPR Gene Therapy Durably Reduces TTR in Patients With ATTR Amyloidosis
Altogether, there was a 91% reduction in median serum TTR at day 28.
Vertex Pauses Diabetes Cell Therapy Trial After Unrelated Patient Deaths
The trial has completed enrollment and all 14 patients have demonstrated islet cell engraftment and endogenous insulin production.
Predictors Not Identified for Return to Prophylaxis Post Fidanacogene Elaparvovec Hemophilia B Gene Therapy
No unifying characteristics were found in a small subset of 6 patients from the BENEGENE-2 trial.
Pfizer’s Hemophilia B Gene Therapy Approved as Beqvez in Canada
The FDA and EMA have accepted marketing authorization applications and decisions are pending.
Solid’s Next-Generation DMD Gene Therapy Cleared for First-in-Human Trial
The AAV-SLB101 capsid used in SGT-003 may yield increased transgene expression and improved safety compared with first-generation AAV gene therapies.
After Exa-Cel: Exploring the Next Wave of CRISPR Gene Editing Strategies
The approval of exa-cel, the first gene editing therapy, may herald a new era in developing gene editing strategies.
Phase 3 Trial of Gene Modifier Therapy for Retinitis Pigmentosa Ready For 2024
Ocugen and the FDA have agreed on trial design aspects including end point, patient enrollment strategy, and study duration.
MDA 2024 to Focus on Patient Experience in Gene Therapy, Nonviral Vectors, and More
CGTLive spoke with Donald Wood, PhD, president and CEO of the MDA while looking ahead to its March conference.
Some Dose-Dependent Neurological Benefits Seen With AMT-130 Huntington Disease Gene Therapy
uniQure plans to initiate regulatory discussions in the US and Europe in the first quarter of 2024.
Luspatercept Improves Symptoms in Nontransfusion Dependent β-Thalassemia
Data from an analysis of patients from the BEYOND study were presented at ASH 2023.
FDA Blocks CARsgen’s 3 Clinical Stage CAR-Ts Over CMC Concerns
Clinical holds have been placed on BCMA-targeted CT053, GPRC5D-targeted CT071, and Claudin18.2-targeted CT041.
Excision Seeks to Suppress HIV Viral Replication With EBT-101 CRISPR Gene Therapy
December is HIV/AIDs Awareness Month and CGTLive is taking a look at Excision's first-in-human CRISPR therapy trial for people with HIV-1.
Most Patients With β-Thalassemia Achieve Transfusion Independence With Exa-Cel Therapy
Exa-cel was recently approved for treating sickle cell disease under the name Casgevy.
Low FVIII Expression After Val-Rox Gene Therapy Maintains Some Hemostatic Benefits
An analysis of participants from GENEr8-1 who had low transgene-derived Factor VIII expression was presented at ASH 2023.
Gene-Edited Cell Therapy Improves Sickle Cell, Transfusion-Dependent Thalassemia Phenotypes
Rabi Hanna, MD, presented updated data from the RUBY and EdiThal trials at ASH 2023.