University of California, Berkeley

CRISPR Gene Editing Breakthrough Saves Baby with Ultra-Rare Metabolic Disorder

7 days ago

• Doctors at Children's Hospital of Philadelphia successfully treated a baby with severe CPS1 deficiency using a personalized CRISPR base-editing therapy, marking a first-of-its-kind approach for this rare metabolic disorder. • The experimental treatment, developed within just six months of diagnosis, corrected the infant's specific genetic mutation by delivering edited DNA to liver cells via lipid nanoparticles, allowing him to reduce medication and process more dietary protein. • This breakthrough demonstrates the potential for creating customized gene therapies for millions with rare genetic diseases, with researchers suggesting costs comparable to liver transplantation and possibilities for treating numerous other conditions.

Glass House Brands Partners with UC Berkeley for Groundbreaking Hemp Research Initiative

11 days ago

• Glass House Brands has established a research collaboration with UC Berkeley to explore hemp-related applications, including medicinal product development, genetics improvement, and AI automation for cultivation. • The partnership will investigate potential drug development targeting coronaviruses, pain, sleep, inflammation, and cirrhosis, while also evaluating evidence-based approaches to hemp policy and regulation. • With a newly acquired hemp license and R&D greenhouse capacity to produce 240,000 pounds of biomass annually, Glass House positions itself to become one of the largest hemp cultivators in the United States.

Tune Therapeutics Secures $175M to Advance Epigenetic Editing for Hepatitis B

4 months ago

• Tune Therapeutics has raised $175 million in a Series B funding round to advance its epigenetic editing therapies, particularly focusing on chronic hepatitis B treatment. • The lead therapy, Tune-401, aims to silence viral DNA in infected cells, offering a novel approach compared to existing treatments that target the virus directly. • Clinical trials for Tune-401 have commenced in New Zealand and Hong Kong, marking a significant milestone in the development of epigenetic editing for chronic diseases. • The funding will also support the expansion of Tune Therapeutics' platform capabilities and the development of additional gene, cell, and regenerative therapy programs.

Bright Minds Biosciences Appoints Stephen Collins as Chief Medical Officer to Advance CNS Therapies

5 months ago

• Bright Minds Biosciences has appointed Stephen D. Collins, M.D., Ph.D., as Chief Medical Officer, effective immediately, to spearhead the development of novel CNS therapies. • Dr. Collins brings extensive experience in drug development and clinical expertise in epilepsy and other CNS disorders, aligning with Bright Minds' focus on unmet medical needs. • Bright Minds is currently evaluating BMB-101, a selective 5-HT2C agonist, in a Phase 2 clinical trial for Absence Epilepsy and Developmental Epileptic Encephalopathy (DEE). • Dr. Collins's prior experience includes leadership roles at Biscayne Neurotherapeutics, Ovation Pharmaceuticals, and Johnson & Johnson, contributing to the development of multiple approved therapies.

Bright Minds Biosciences Appoints Stephen Collins as Chief Medical Officer to Advance Epilepsy Therapies

5 months ago

• Bright Minds Biosciences has appointed Stephen Collins, M.D., Ph.D., as Chief Medical Officer, effective immediately, to lead clinical development efforts. • Dr. Collins brings extensive experience in CNS drug development, particularly in epilepsy, and will guide the Phase 2 clinical trial of BMB-101. • BMB-101, a selective 5-HT2C agonist, is currently under evaluation in the BREAKTHROUGH Study for adult patients with Absence Epilepsy and DEE. • Bright Minds Biosciences focuses on developing innovative treatments for neurological and psychiatric disorders, addressing unmet needs in CNS conditions.

Opus Genetics to Present 6-Month Data on OPGx-LCA5 Gene Therapy for LCA5-Associated Inherited Retinal Disease

6 months ago

• Opus Genetics will share detailed 6-month efficacy and safety data on OPGx-LCA5, a gene therapy for LCA5-associated inherited retinal disease, at a virtual KOL event. • OPGx-LCA5 uses an AAV8 vector to deliver a functional LCA5 gene to the outer retina, potentially addressing the unmet need in this early-onset severe retinal dystrophy. • The Phase 1/2 clinical trial at the University of Pennsylvania is evaluating the safety and preliminary efficacy of OPGx-LCA5 in patients with LCA5 gene mutations. • Key opinion leaders will discuss the potential of Opus Genetics’ therapeutic approach and the next steps in the development program for OPGx-LCA5.

FDA Updates Bioresearch Monitoring Technical Specifications for NDAs and BLAs

7 months ago

• The FDA has released an updated Bioresearch Monitoring (BIMO) Technical Conformance Guide, version 3.1.1, providing detailed specifications for the BIMO section of New Drug Applications (NDAs) and Biologics License Applications (BLAs). • The guide standardizes the structure and format of clinical trial data submissions, ensuring transparency and facilitating FDA's review process, particularly during BIMO inspections. • Key areas covered in the document include clinical study-level information, subject-level data line listings by clinical site, and a summary-level clinical site data set, all crucial for data integrity assurance. • The technical document outlines how to categorize and document the location of clinical trial data, aiding FDA investigators in efficiently locating raw data across various sites, including clinical sites and CROs.

Anixa Biosciences Bolsters Clinical Development with Suyasha Gupta Appointment

8 months ago

• Anixa Biosciences has appointed Suyasha Gupta as Senior Director of Clinical Development to enhance its oncology programs. • Gupta will oversee ongoing Phase 1 trials for breast cancer vaccine and ovarian cancer CAR-T therapy, plus an upcoming Phase 2 breast cancer vaccine trial. • Gupta's experience includes managing late-stage oncology trials at Genentech, focusing on immunotherapy and biomarker strategies. • Anixa anticipates Gupta's expertise will accelerate the development of innovative cancer therapies, particularly in late-stage trials.

AI Tool Predicts Responses to Cancer Therapy Using Single-Cell Omics

a year ago

A groundbreaking AI tool named PERCEPTION has been developed to predict patient responses to cancer drugs at single-cell resolution, offering a new approach to personalized cancer treatment by utilizing rich information from single-cell omics.