ISSCR News
Now Available: Free, On-Demand Course “Applying the ISSCR Standards for Research”
The International Society for Stem Cell Research (ISSCR) and STEMCELL Technologies today announced the launch of Applying the ISSCR Standards for Research, a free, on-demand course designed to help scientists integrate the ISSCR Standards for Human Stem Cell Use in Research into their work. The course provides practical guidance to strengthen rigor, reproducibility, and responsible use of human stem cells across the research pipeline.
Member Spotlight: Yasuhiro Takashima, MD, PhD
[ISSCR membership] allows me to build friendships with stem cell researchers from around the world.
Retinal Organoid Platform Identifies Biomarkers and Affords Genetic Testing for Retinal Disease
Leber Congenital Amaurosis (LCA) is an inherited retinal disease leading to severe vision impairment from early infancy, affecting 2-3 out of every 100,000 newborns. LCA is caused by variants in certain genes from which proteins essential for retinal cell function and survival are made. Mutations in over 20 different genes can cause LCA and in each of those genes, hundreds of different changes, known as genetic variants, can occur. Importantly, while some of these genetic variants are benign and do not compromise function, other variants cause dysfunction or complete loss of function. Further, for some of these variants, which are called variants of uncertain significance (VUS), it is currently not known if they are disease causing or benign.
The ISSCR Responds to FDA’s Draft Guidance on Innovative Designs for Clinical Trials of Cellular and Gene Therapy Products in Small Populations
On 24 November, the ISSCR submitted comments on the Food and Drug Administration's draft guidance for Innovative Designs for Clinical Trials of Cellular and Gene Therapy Products in Small Populations. ISSCR appreciates FDA’s willingness to consider alternative trial designs for cellular and gene therapy products. To support this effort, ISSCR requests additional guidance on managing manufacturing changes within adaptive or master protocols; decentralized trials for small populations; long-term follow-up; using surrogate endpoints and biomarkers; and trial design selection.
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