The Simons Foundation Autism Research Initiative (SFARI) is pleased to announce that it intends to fund 17 grants in response to the 2021 Genomics of ASD: Pathways to Genetic Therapies request for applications (RFA).
Grants funded through this RFA are intended to leverage current knowledge about risk genes for autism spectrum disorder (ASD) into the development of new genetic therapies.
Applications in response to this RFA were sought in three broad areas: (1) risk gene discovery and systems biology, (2) functional analysis of variants associated with ASD risk genes and (3) gene-targeted therapies. Proposals that span the different focus areas were encouraged, as were collaborations between academic and industry partners. Furthermore, SFARI encouraged proposals that focused on a subset of 50 genes from the SPARK gene list; these genes were selected, for a variety of different reasons, as strong candidates for the development of translational programs.
SFARI intends to provide approximately $17 million in funding over the next three years to 17 investigators as part of this program.
“Congratulations to all of the investigators and their collaborators who’ve received these awards,” says Kelsey Martin, director of SFARI and the Simons Foundation Neuroscience Collaborations. “The autism research community has made great strides in the identification of hundreds of risk genes over the past decade, and our hope now is that this knowledge can be translated into the development of treatments for genetically defined subtypes of the condition.”
The projects that were selected for funding focus on several different risk genes and conditions, including GRIN disorders, Rett syndrome and SLC6A1-related autism disorder. A variety of different approaches and methods will be used, including high-throughput screens to ascertain the functional effects of autism variants, and the development of antisense oligonucleotide and adenoviral vector-based gene therapies.
SFARI senior scientist Alan Packer adds, “We look forward to seeing the outcomes of these studies over the next few years and gaining valuable insights that will help accelerate progress toward new therapies for individuals with autism and their families.”
The projects that SFARI intends to fund are:
Allison Bradbury, Ph.D. (Nationwide Children’s Hospital)
Optimization and validation of gene therapy using a patient-specific mouse model of SLC6A1-related disorders
Samouil Farhi, Ph.D. and Ralda Nehme, Ph.D. (Broad Institute of Massachusetts Institute of Technology and Harvard University)
Multimodal fingerprinting of cellular phenotypes associated with autism risk variants
Joseph Gleeson, M.D. (University of California, San Diego)
Stem cell models to assess antisense oligonucleotides as novel therapeutics for autism
Nael Nadif Kasri, Ph.D. (Radboud University Medical Center)
A human stem cell-based neuronal network platform for functional validation of autism-linked variants
Christopher J. R. Loewen, Ph.D. (University of British Columbia)
High-throughput autism variant functional testing using genetic interaction technologies in model systems
Wei Niu, Ph.D. and Jack M. Parent, M.D. (University of Michigan)
Antisense oligonucleotide therapy of X-linked protocadherin 19-related autism and epilepsy
Pablo Perez-Pinera, M.D., Ph.D. and Thomas Gaj, Ph.D. (University of Illinois at Urbana-Champaign)
Development of a CRISPR base editing gene therapy for Rett syndrome
Jonathan Sebat, Ph.D. (University of California, San Diego)
Platforms to accelerate development of antisense oligonucleotide therapeutics for haploinsufficiencies associated with autism
Rosanna Weksberg, M.D., Ph.D. (The Hospital for Sick Children; University of Toronto)
Functional annotation of gene variants in autism risk genes using DNA methylation signatures
- Updates to SFARI’s 2021 requests for grant applications
- SFARI workshop explores challenges and opportunities of gene therapies for autism spectrum disorder
- SFARI 2021 Genomics of ASD: Pathways to Genetic Therapies RFA — Informational session
- Seeing through a forest of SCN2A gene variation
- Making sense out of missense mutations
- A joint variant call-set for all Simons Simplex Collection whole-genome sequences is now available