Bemdaneprocel Moves to Phase 2 Study in Parkinson Disease After Positive Phase 1 Data
Significant improvements in ON time without troublesome dyskinesia were reported with the cell therapy after 1 year, with more apparent effects observed in the high-dose group.
Design Therapeutics' DT-216 Displays Positive Phase 1 Results in Friedreich Ataxia
Data from a multiple-ascending dose study suggest the investigational agent developed by Design Therapeutics was generally well tolerated with dose-related increases in frataxin levels.
NurOwn Cell Therapy for ALS Will Have Its Day in Front of FDA AdComm After All
In November 2022, BrainStorm received a refusal to file letter from the FDA for the biologics license application of NurOwn.
Fabry Disease Gene Therapy Demonstrates Favorable Impact in Phase 1/2 Trial
All dose-escalation patients withdrew from enzyme replacement therapy (ERT) and remain off ERT.
ALS Cell Therapy BLA Refused for Filing by the FDA
BrainStorm received indication from the FDA that it is able to request a Type A meeting to discuss the content of the refusal to file letter.
Upstaza Approved by European Commission for AADC Deficiency
The gene therapy received a positive CHMP opinion in May 2022.
Vutrisiran Approved for hATTR Amyloidosis
The approval came earlier than the recently extended PDUFA date of July 24, 2022.
EMBARK Study Evaluates DMD Gene Therapy
Sarepta also presented updated data from Study 101 of SRP-9001 at MDA 2022.
Mesenchymal Stem Cell Therapy Reduces NfL in Progressive MS
Patients demonstrated a median reduction of 63.5% in CSF NfL levels 6 months after treatment.
Autologous Adipose Stem Cells Safe in Alzheimer Disease, More Efficacy Data Needed
Autologous adipose stem cell data in a small cohort provides a feasibility for future validation studies.
Bone Marrow-Derived Mesenchymal Stem Cells Safe in ALS
ALSFRS-R and FVC values were stable 3 months after treatment but significantly decreased 6 months after treatment, warranting further investigation.
Gene Therapy for Parkinson Disease Demonstrates Safety, Improved Motor Function
From baseline, all participants showed improvements on CGI-Improvement scale scores while all but 1 patient demonstrated improvement on modified Hoehn and Yahr scores.
IND Cleared for Focal Epilepsy Cell Therapy Phase 1/2 Trial
The cell therapy is set to be evaluated in a 2-part, phase 1/2 trial.
Tofersen Fails to Meet Endpoint in Familial ALS Despite Favorable Trends
Tofersen failed to meet its primary end point in change from baseline in ALSFRS-R but differences in total cerebrospinal fluid SOD1 protein and neurofilament light chain were observed.
Presymptomatic Zolgensma Treatment Yields Age-Appropriate Development in SMA
Newly published data continues to demonstrate Zolgensma's efficacy in presymptomatic and symptomatic SMA Type 1.
FDA Accepts IND for Limb-Girdle Muscular Dystrophy Gene Therapy
Asklepios BioPharmaceuticals noted that it plans to initiate dosing for the LION-101 phase 1/2 clinical trial in the first half of 2022.
Gene Therapy for SLC13A5 Deficiency Shows Promise in Preclinical Data
No adverse effects on weight, general activity, or survival in knockout mice and wild type littermates were observed throughout the study.
FDA Clears IND Application for SOD1-ALS Gene Therapy
Apic Bio plans to initiate a phase 1/2 clinical trial in late 2021 or early 2022 as a multi-center, 3-part study to evaluate APB-102 in patients with SOD1-ALS mutations.
Stem Cell Stroke Therapy Demonstrates Safety in Treatment of Ischemic Stroke
Clinically relevant scores of 32 and 44 on the Fugl-Meyer Motor Score were documented in 2 patients who received NSI-566.
Experimental DMD Gene Therapy Phase 3 Trial Launched
The Pfizer candidate PF-06939926 has also received fast track, orphan drug, and rare pediatric disease designations from the FDA.
NurOwn Expanded Access Program Announced
The Expanded Access Program will offer treatment for patients with ALS who have completed the phase 3 clinical trial and will not interfere with data or regulatory timelines.
SRP-5051 Shows Efficacy in DMD Amenable Exon 51 Skipping
Results of the phase 2 study support continued dose escalation of SRP-5051 and further clinical development.
Intrathecal Zolgensma Shows Early Efficacy in SMA
Nearly all patients in this cohort achieved a clinically meaningful >3-point increase during the study period, demonstrating a consistent response to the gene therapy.
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