CIRM awarded Elpida Therapeutics a $3.9 million grant in December 2023 to support a pioneering preclinical study on Charcot Marie Tooth disease type 4J (CMT4J).
gene therapy
Gene therapy trial tests cancer-killing virus in brain tumors
Dr. Noriyuki Kasahara from UCSF has been awarded $11.8 million by the California Institute for Regenerative Medicine (CIRM) to target newly diagnosed high-grade gliomas. This groundbreaking clinical trial aims to treat high-grade gliomas, one of the most challenging brain cancers to manage. These aggressive cancers develop in the brain or spinal cord and are notoriously … Continue reading Gene therapy trial tests cancer-killing virus in brain tumors
Equipping California nurses with skills to support cell and gene therapy patients
Regenerative medicine is a rapidly evolving field. As more therapies reach commercialization and clinical trial stages, there remains an immediate need to equip direct patient care nurses with skills to support patients on complex clinical trials as well as novel standard-of-care therapies. To address this need and continue efforts to build a regenerative medicine workforce … Continue reading Equipping California nurses with skills to support cell and gene therapy patients
Update on SCID patient enrolled in CIRM-funded gene therapy trial
Photo credit: UCSF Hataalii Tiisyatonii Begay (HT) is paving the road for newborns with SCID. When HT was born in 2018 in a remote part of the Navajo nation, he was quickly diagnosed with a rare and -usually fatal- condition. Today, thanks to a therapy developed at UCSF and funded by CIRM, he’s a healthy … Continue reading Update on SCID patient enrolled in CIRM-funded gene therapy trial
Reminder! Apply now for discovery stage stem cell and gene therapy research funding
The California Institute for Regenerative Medicine (CIRM) is seeking applications for its next round of Quest Awards (DISC2) for discovery stage research. Applications are due August 2nd, 2022, at 2:00 PM PDT. Please visit the CIRM website for full details. The purpose of the Quest Awards is to promote the discovery of promising new stem cell-based or gene therapy … Continue reading Reminder! Apply now for discovery stage stem cell and gene therapy research funding
An experimental gene therapy with a hairy twist
In October 2019, 20-year-old Jordan Janz became the first person in the world to receive an experimental therapy for cystinosis. Cystinosis is a rare genetic disorder characterized by the accumulation of an amino acid called cystine in different tissues and organs of the body including the kidneys, eyes, muscles, liver, pancreas, and brain. This accumulation … Continue reading An experimental gene therapy with a hairy twist
Creating a ‘bespoke’ approach to rare diseases
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Up until recently the word “bespoke” meant just one thing to me, a hand-made suit, customized and fitted to you. There’s a street in London, Saville Row, that specializes in these suits. They’re gorgeous. They’re also very expensive and so I thought I’d never have a … Continue reading Creating a ‘bespoke’ approach to rare diseases
UCLA gene therapy offers children with LAD-1 a new chance at living a normal life
THIS BLOG IS ALSO AVAILABLE AS AN AUDIO CAST Photo courtesy of Tamara Hogue/UCLA Broad Stem Cell Research Center Leukocyte adhesion deficiency type 1 (LAD-1) is a rare pediatric disorder that causes the immune system to malfunction, resulting in recurrent, often severe, bacterial and fungal infections as well as delayed wound healing. This is because of a missing protein that would normally … Continue reading UCLA gene therapy offers children with LAD-1 a new chance at living a normal life
Celebrating a young life that almost wasn’t
Often on the Stem Cellar we feature CIRM-funded work that is helping advance the field, unlocking some of the secrets of stem cells and how best to use them to develop promising therapies. But every once in a while it’s good to remind ourselves that this work, while it may often seem slow, is already … Continue reading Celebrating a young life that almost wasn’t
CIRM funded trial may pave way for gene therapy to treat different diseases
Image Description: Jordan Janz (left) and Dr. Stephanie Cherqui (right) According to the National Organization for Rare Disorders (NORD), a disease is consider rare if it affects fewer than 200,000 people. If you combine the over 7,000 known rare diseases, about 30 million people in the U.S. are affected by one of these conditions. A … Continue reading CIRM funded trial may pave way for gene therapy to treat different diseases