Going Public: Doudna’s Dream Team Launches Groundbreaking Sickle Cell Trial

A publicly funded consortium of University of California (UC) researchers and clinicians is one big step closer to realizing a decade-long plan to launch a CRISPR-based gene-editing trial for sickle cell disease (SCD). Last week, the team, led by 2020 Nobel laureate Jennifer Doudna, PhD, announced...

Novel Gene Therapy Trial for Sickle Cell Disease Launches

The study uses CRISPR to correct sickle cell mutation and aims to free patients of the disease.

Neurona Therapeutics Receives FDA’s Regenerative Medicine Advanced Therapy (RMAT) Designation for NRTX-1001 in Focal Epilepsy

– RMAT designation follows positive data update from the ongoing Phase 1/2 clinical trial of NRTX-1001 cell therapy, demonstrating a favorable safety profile to date and the potential to significantly reduce seizure frequency in drug-resistant epilepsy – 60% (3 of 5 subjects) who received a single...

UCSF Treats First Patient with Homegrown Chimeric Antigen Receptor (CAR) T-cell Therapy

This month marked an important milestone for UCSF faculty and clinicians working to take on the challenge of glioblastoma multiforme (GBM). They treated their first patient with a promising chimeric antigen receptor (CAR) T-cell therapy that was fully conceived, designed, optimized and manufactured...

Gene Therapy Is Halting Cancer. Can It Work Against Brain Tumors?

Grant of up to $11 million will fund a clinical trial at UCSF that uses a smarter new CAR-T guided by precision technology.

Fetal therapy offers hope for unborn patients suffering from the rare genetic disease Hunter Syndrome

Some parents-to-be have to face the terrifying news that their unborn child has been diagnosed with a life-threatening genetic disorder. But thanks to a medical team at a Bay Area hospital, there is amazing new hope in a fetal therapy that may spare their unborn child serious outcomes.   

New CRISPR Center Brings Hope for Rare and Deadly Genetic Diseases

CRISPR collaboration combines expertise from three UC schools to scale treatment for diseases that industry has largely passed by – until now.

The Surgeon Set on Curing Genetic Diseases Before Birth

Five-year-old Elianna loves running around with her big brother in their yard on the Hawaiian island of Kauai. She isn’t afraid to get dirty, is learning to ride a bike, and has her doting grandfather wrapped around her little finger. It’s a delightfully ordinary childhood – but one that almost...

Two Daughters, a Rare Blood Disorder and a Fairytale Cure

With colorful headphones and eyes glued to her favorite Phineas and Ferb Disney movie on the family iPad, Ada reclined in her hospital bed tethered to a bag of medications and syringes of blood. She was in the middle of a gene therapy transplant – an infusion of her own blood cells that had been...

NIH Innovator Awards

The New Innovator Award is part of the NIH Common Fund's High-Risk, High-Reward Research program and supports exceptionally creative early career investigators proposing innovative, high-impact research.