UCLA Receives Major Funding for Groundbreaking Gene Therapy
Research
In an exciting development, the team at UCLA has been awarded a significant $5.8 million translational research grant from the California Institute for Regenerative Medicine (CIRM). This funding will support pivotal work on gene therapy aimed at treating Angelman syndrome (AS), a neurodevelopmental disorder that results in severe cognitive and physical impairment.
About the Research
Dr. Roger Hollis, a project scientist at UCLA, is leading the charge alongside Dr. Donald Kohn, a prominent figure in the field of gene replacement therapies utilizing blood stem cells. Their innovative approach involves modifying a patient’s own blood stem cells to integrate a healthy copy of the UBE3A gene, which functions differently or is absent in individuals affected by AS.
Innovative Approaches to Treatment
The initial data for this groundbreaking research has shown outstanding results. Funded by TransformaTx Biotherapeutics and the Foundation for Angelman Syndrome Therapeutics (FAST), the studies conducted on adult mouse models exhibited complete symptomatic correction, signifying the potential effectiveness of this therapy in humans.
Expert Insights from the UCLA Team
This program has been garnering positive animal data results, reinforcing its promise as a revolutionary treatment option. Dr. Kohn expressed enthusiasm about collaborating with TransformaTx Biotherapeutics and FAST to move this investigational gene therapy forward into clinical trials, offering more options for patients living with AS.
Preparations for Clinical Trials
Chris Luthers, the lead scientist in molecular biology at UCLA overseeing the HSC-GT development, presented findings from both in vitro and in vivo studies at a recent Global Summit on Angelman syndrome. The new funding will facilitate the preparation of a pre-IND package submission to the U.S. Food and Drug Administration, marking an essential step toward initiating a Phase 1 clinical trial focused on evaluating the therapy’s safety and efficacy.
Community Support Drives Research Forward
Dr. Allyson Berent, chief development officer at TransformaTx Biotherapeutics and chief science officer of FAST, emphasized that securing this crucial funding is a major advancement for the research team. The success achieved thus far stands as a testament to the unwavering dedication of the scientific team and the support from the AS community, driving progress for this promising program.
Media Contact: Meghan Edberg, Director of Public Engagement (630-852-3278)
Frequently Asked Questions
What is Angelman syndrome?
Angelman syndrome is a neurogenetic disorder characterized by developmental delays, problems with balance and movement, and occasional seizures. It significantly impacts cognitive and physical abilities.
Who is leading the research at UCLA?
The research is being led by Dr. Roger Hollis, in collaboration with Dr. Donald Kohn, both of whom are experts in the field of gene therapies for genetic disorders.
What is the importance of the UBE3A gene?
The UBE3A gene plays a critical role in the normal functioning of the nervous system. In individuals with Angelman syndrome, this gene is typically not expressed in the neurons, leading to the associated symptoms.
What are the next steps for this research?
The UCLA team is preparing for a pre-IND submission to the FDA, which will facilitate the transition towards initiating Phase 1 clinical trials to test the gene therapy in humans.
How can the community support this research?
Community support is invaluable for research initiatives like this. Contributions can come in many forms, including donations, participation in awareness events, and advocacy for funding for ongoing research efforts.
