Brainstorm Cell Therapeutics

DelveInsight's report on the Gene and Cell Therapies Targeting CNS Disorders market forecasts growth to 2034, highlighting key companies and therapies, including Bayer AG's Phase Ib clinical trial success for Parkinson's disease and uniQure's positive trends in Huntington's disease trials. The market, valued at approximately USD 1 billion in 2021, is expected to surge due to increasing disease prevalence and pipeline product launches.

DelveInsight's 'Multiple Sclerosis Pipeline Insight 2024' report highlights 75+ companies developing 80+ therapies, including promising candidates like GA Depot, Remibrutinib, and IMU 838. Key events include Immunic's positive Phase 3 ENSURE trial interim analysis, Roche's FDA approval for Ocrevus injectable, and Sanofi's mixed results for tolebrutinib in Phase III trials. The report covers global pipeline stages, product types, molecule types, mechanisms of action, and routes of administration.

Jason McCarthy of Maxim Group maintains a Buy rating on VistaGen Therapeutics (VTGN) with a $12.00 price target, citing successful Phase 3 trials for fasedienol in treating social anxiety disorder and a robust financial position with $97.6 million in cash reserves.

Biomarker data indicate ALS patients may benefit from long-term NurOwn treatment. A Phase 3b trial design was also presented, aiming to confirm NurOwn's efficacy in early-stage ALS patients.

BrainStorm Cell Therapeutics Inc. announced participation in the 2024 Maxim Healthcare Virtual Summit, where CEO Chaim Lebovits will discuss the planned Phase 3b clinical trial for NurOwn®, the company's ALS treatment. The summit, from October 15 to 17, will feature presentations and discussions on healthcare trends, accessible via M-Vest.

The ALS drug development pipeline is rich with promising therapies targeting TDP-43 pathology, including Biogen’s BIIB-105, AbbVie/Calico’s ABBV-CLS-7262, Denali’s DNL-343, and QurAlis’ QRL-201. Trials range from Phase 1 to Phase 3, with completion dates extending to 2026. These therapies aim to slow ALS progression by addressing TDP-43 mislocalization and aggregation, a hallmark of ALS pathology. The article also highlights the importance of clinical trials in advancing ALS treatment and the potential for FDA approval of these therapies.

Autophagy, crucial for neuronal health, is impaired in ALS, making its regulation a potential therapeutic target. Various drugs, including Rapamycin, Trehalose, Lithium, and others, show promise in enhancing autophagy and improving ALS symptoms in animal models, though clinical efficacy varies. Gene therapies and stem cell transplantation also offer hope, with ongoing research into their safety and effectiveness for ALS treatment.