UNIVERSITY OF CALIFORNIA, DAVIS

Promising Alzheimer's Vaccine Targeting Tau Protein Advances Toward Human Trials

a month ago

• University of New Mexico researchers have developed a vaccine that generates antibodies against pathological tau protein (pT181), showing efficacy in both mice and non-human primates with strong immune responses. • The vaccine uses virus-like particles (VLPs) as a delivery platform, requiring only one primary shot and two boosters without adjuvants, demonstrating safety and durability in preclinical studies. • Researchers are now seeking funding to advance to Phase 1 human clinical trials, potentially offering a more effective approach than current amyloid beta-targeting treatments that show only modest effects on Alzheimer's progression.

Phase 3 Trial Shows Promising Results for Novel Encapsulated Cell Therapy in MacTel Treatment

3 months ago

• Phase 3 clinical trials of NT-501, an encapsulated cell therapy delivering CNTF, demonstrated significant reduction in disease progression for macular telangiectasia type 2 patients, with up to 52% reduction in ellipsoid zone loss. • The innovative implantable device, developed by Neurotech, maintains long-term viability with CNTF production documented for up to 14.5 years, offering a potential alternative to frequent intravitreal injections. • FDA review of the therapy is currently underway with a PDUFA date set for March 18, 2025, marking a potential breakthrough in MacTel treatment.

Solid Biosciences' SGT-003 Gene Therapy Shows Promise in Duchenne Muscular Dystrophy Trial

4 months ago

• Solid Biosciences reported positive initial data from its Phase 1/2 INSPIRE DUCHENNE trial of SGT-003, a gene therapy candidate for Duchenne muscular dystrophy (DMD). • The trial showed an average microdystrophin expression of 110% in the first three patients, along with improvements in biomarkers indicating muscle health and resilience. • SGT-003 was well-tolerated in the first six participants, with adverse events typical of AAV gene therapy and no serious safety concerns reported. • Solid Biosciences plans to meet with the FDA to discuss a potential accelerated approval pathway for SGT-003 based on these encouraging early results.

Neurogene's Rett Syndrome Gene Therapy Trial Pauses High Dose After Patient Death

6 months ago

• A patient with Rett syndrome died after receiving a high dose of Neurogene's experimental gene therapy, NGN-401, triggering a safety review. • The FDA is allowing Neurogene to continue the Phase 1/2 clinical trial with a lower dose of NGN-401, which has shown promising early results. • The adverse event underscores the risks associated with high doses of AAV vectors in gene therapy, particularly regarding immune responses. • Neurogene plans to update the clinical trial protocol to remove the high dose and expects to resume dosing in the low-dose cohort after revisions.

CRISPR-Cas9 Reduces VEGF Levels in Monkeys, Shows Retinal Toxicity at High Doses

8 months ago

• CRISPR-Cas9 targeting VEGFA delivered via AAV vectors reduces VEGF levels in monkeys, potentially treating choroidal neovascularisation (CNV). • Low AAV doses (6 x 1011 vg/eye) led to a 33% reduction in VEGF protein concentrations and decreased CNV severity. • High AAV doses (6 x 1012 vg/eye) resulted in significant retinal toxicity, including subfoveal deposits and outer retinal thinning. • Retinal damage appears linked to high Cas9 expression or AAV vector dose, not VEGFA gene editing itself, suggesting optimization is needed.

CNP-104 Shows Promise in Phase 2a Trial for Primary Biliary Cholangitis

8 months ago

• CNP-104 demonstrated a statistically significant decrease in liver stiffness compared to placebo in patients with primary biliary cholangitis (PBC). • The investigational therapy also showed favorable changes in pathogenic CD4 T cell populations and tolerance-inducing CD8 T cells. • CNP-104 was safe and well-tolerated, with all drug-related adverse events reported as mild during the 120-day primary study period. • These results suggest CNP-104 has the potential to halt disease progression, offering a transformational advancement for PBC treatment.

Community Trial to Assess Early Start Denver Model (ESDM) for Young Children with Autism

8 months ago

• A cluster randomized controlled trial will compare the effectiveness of the Early Start Denver Model (ESDM) versus usual early behavioral intervention (EBI) for young children with autism. • The study will recruit 300 child/caregiver dyads across 20 community-based agencies (CBAs) in the US, randomizing regions within CBAs to either ESDM or EBI. • The primary outcome will be child social communication and language development, with secondary outcomes including adaptive behavior, cognitive gains, and caregiver competence. • Implementation outcomes, including acceptability, appropriateness, and feasibility of ESDM, will also be assessed to inform future scale-up efforts.

Condoliase (SI-6603) Shows Promise in Phase 3 Trials for Lumbar Disc Herniation

8 months ago

• SI-6603, an investigational treatment, significantly improved leg pain compared to sham in U.S. and placebo in Japan at Week 13 in Phase 3 trials. • The studies involved intradiscal injections of SI-6603 and demonstrated statistically significant changes from baseline in worst leg pain. • Common adverse events included spinal MRI abnormalities and back pain, with no treatment-related serious adverse events reported in the U.S. study. • These findings support SI-6603's potential as a therapeutic option for radicular leg pain associated with lumbar disc herniation.

Capricor Therapeutics Advances Deramiocel for Duchenne Muscular Dystrophy Cardiomyopathy

8 months ago

• Capricor Therapeutics plans to file a Biologics License Application (BLA) with the FDA for deramiocel to treat Duchenne muscular dystrophy (DMD) cardiomyopathy. • The BLA will be supported by cardiac data from Phase 2 HOPE-2 and HOPE-2 OLE trials, compared with natural history data. • Capricor has initiated its rolling submission process with the FDA for deramiocel, with completion expected by the end of 2024. • Deramiocel has shown immunomodulatory, antifibrotic, and regenerative actions in dystrophinopathy and heart failure in clinical studies.

NIH-Funded Study to Investigate Type 1 Diabetes' Impact on Children's Brain Development

8 months ago

• A nationwide longitudinal study, funded by a $2.7 million NIH grant, will explore the effects of type 1 diabetes on brain development in children. • The multi-site study will involve 12 research centers across the U.S. and recruit over 1,000 children newly diagnosed with type 1 diabetes. • Researchers aim to identify factors that may accelerate or mitigate the risk of brain-related complications in children with type 1 diabetes. • The study will use fMRI and collect data on academic performance, memory, attention, and neurobehavioral health to refine clinical guidelines.

Ibogaine Shows Promise in Reducing PTSD and TBI Symptoms in Veterans

a year ago

• A recent study published in Nature Medicine indicates that ibogaine, a psychedelic drug, significantly reduced PTSD, anxiety, and depression symptoms in combat veterans. • The research, conducted by Stanford University, observed improvements in cognitive function among veterans with traumatic brain injuries (TBI) following ibogaine treatment. • The study involved 30 special forces veterans who received ibogaine treatment at a clinic in Mexico, highlighting the potential of psychedelics in treating trauma-related conditions. • Further research is needed to replicate these results in broader populations and to explore the mechanisms through which ibogaine impacts brain function and mental health.

Izotropic Files Pre-Submission Application With U.S. FDA for Breast CT Imaging System

5 years ago

Izotropic Corporation has taken a significant step towards market approval for its Breast CT Imaging System by filing a Pre-Submission Application with the U.S. FDA. This move marks a pivotal moment in the company's journey to revolutionize breast cancer imaging, aiming to provide a less painful and more efficient diagnostic tool compared to traditional mammography.