Awards by Year
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RETT SYNDROME RESEARCH TRUST AWARDS BY YEAR TOTAL AWARDS $62 MILLION (2008-2021 YTD) 2021 (to date) TOTAL AWARDS $1,469,143 Antonio Bedalov / Kyle Fink Jackson Laboratories Fred Hutchinson Cancer Research Institute / Testing of siRNA compounds from Khvorova University of California Davis lab for MECP2 Duplication Syndrome Reactivation of MECP2 $362,930 $1,090,919 Bryce Reeve, PhD Duke University School of Medicine Development of the Observer-Reported Communication Ability (ORCA) for Rett Syndrome $15,294 2020 TOTAL AWARDS $1,299,972 DSG Bryce Reeve, PhD Development of the Rett Syndrome Global Registry Duke University School of Medicine $693,000 Development of the Observer-Reported Communication Ability (ORCA) for Rett Syndrome James Wilson, MD, PhD $72,225 University of Pennsylania MECP2 gene therapy for Rett Syndrome Ciitizen $380,686 Pilot Study for Digital Natural History Study $34,885 Clinical Trial Consortium David Lieberman, MD, PhD Sasha Djukic, MD, PhD Boston Children’s Hospital Albert Einstein College of Medicine $94,176 Support for continuing work at the Rett Syndrome Center $25,000 Due to the global pandemic and the ensuing fundraising uncertainties we were cautious in taking on additional commitments. Furthermore we undertook a detailed analysis of our portfolio and were able to reduce our commitments by $6 million. This reduction allows us to focus our resources on curative projects with the greatest likelihood of success in the nearer term. 2019 TOTAL AWARDS $8,570,181 Adrian Bird, PhD / Michael Greenberg, PhD / James Wilson, MD, PhD Gail Mandel, PhD University of Pennsylvania University of Edinburgh / Harvard University / MECP2 gene therapy for Rett Syndrome Oregon Health and Sciences University $765,607 MECP2 Consortium $3,359,054 2 James Wilson, MD, PhD Joseph Jacobson, PhD University of Pennsylvania Massachusetts Institute of Technology MECP2 gene therapy for Rett Syndrome, vector production Correction of MECP2 mutations with engineered ScCas 9 $37,999 base editors $50,000 Stuart Cobb, PhD / Chris Sibley, PhD University of Edinburgh The Jackson Laboratory RNA trans-splicing therapy in Rett Syndrome Generation and phenotypic assessment of mouse models for $235,950 Rett Syndrome $417,690 Harvard Stem Cell Institute Support for development of patient derived induced pluripotent Coriell Institute stem cell lines Rett Syndrome biorepository $101,912 $135,000 Michael Elowitz, PhD Emerald Innovations California Institute of Technology Passive monitoring of Rett patients with Emerald A system for dosage-independent control of MECP2 expression $164,670 in Rett Syndrome gene therapy $212,374 Beth McCormick, PhD University of Massachusetts Medical School Peter Glazer, PhD / Mark Saltzman PhD Microbiome study for the advancement of novel Yale University nutritional supplements PNA nanoparticles for gene editing of Rett Syndrome $520,316 $275,000 Sasha Djukic, MD, PhD Alanna Schepartz, PhD Albert Einstein School of Medicine Yale University Support for continuing work at the Rett Syndrome Center Evaluating cell-permeant miniature proteins (CPMPs) as a strategy $75,000 for delivering functional MECP2 into model cells and neurons $297,716 Miscellaneous Pilot Studies $135,522 Joost Gribnau, PhD Erasmus Medical Center Ronald Cohn, PhD The Hospital for Sick Children Human in vitro models for X chromosome reactivation Interrogation of genome editing strategies as a therapeutic $401,000 modality for MECP2 Duplication Syndrome Antonio Bedalov, PhD $570,000 Fred Hutchinson Cancer Research Center Anastasia Khvorova, PhD Mouse model maintenance University of Massachusetts Medical School $20,000 Development of siRNA based compounds to potently silence Thorsten Stafforst, PhD MECP2 towards the treatment of MECP2 Duplication Syndrome University of Tubingen RNA editing for MECP2 mutations via RESTORE $359,856 3 2018 TOTAL AWARDS $9,956,283 Jonathan Watts, PhD / Scot Wolfe, PhD / James Wilson, MD, PhD Eric Sontheimer, PhD / Anastasia Khvorova, PhD University of Pennsylvania University of Massachusetts Medical School Gene Therapy Consortium Vector Core RNA and genome editing for treatment of Rett Syndrome $131,243 $2,403,735 Allan Jacobson, PhD / Jonathan Watts, PhD Guoping Feng, PhD / Feng Zhang, PhD / University of Massachusetts Medical School Robert Desimone, PhD Read-through of premature termination codons for treatment Massachusetts Institute of Technology / Broad Institute / Rett Syndrome Harvard University $323,000 RNA-editing as a gene therapy approach for Rett Syndrome Antonio Bedalov $2,332,000 Fred Hutchinson Cancer Research Institute Beam Therapeutics Reactivation of MECP2 Developing a pre-clinical DNA base editing program to precisely correct $38,000 the genetic cause of Rett Syndrome in the central nervous system Clinical Trial Consortium $1,870,660 David Lieberman, MD, PhD John Sinnamon, PhD Boston Children’s Hospital Oregon Health and Science University $74,792 New editing enzymes for RNA Laurel Joy Gabard-Durnam, PhD $345,000 Harvard University Peter Beal, PhD Post Doctoral Fellowship, Autism Science Foundation University of California, Davis $17,500 New molecular tools for directed editing of MECP2 mutations associated with Rett Syndrome Hassan Ghasemzadeh, PhD Washington State University $563,870 Pilot study to examine gait patterns in Rett Syndrome Stuart Cobb, PhD / Adrian Bird, PhD $10,000 University of Edinburgh Gene Therapy Consortium 2.0 Sasha Djukic, MD, PhD Albert Einstein College of Medicine $653,856 Support for continuing work at the Rett Syndrome Center Stuart Cobb, PhD $75,000 University of Edinburgh Purchase of qPCR machine Huda Zoghbi, MD, PhD Baylor College of Medicine $13,945 A forward genetic screen to identify druggable modulators of Andrea Cerase, PhD MECP2 levels Queen Mary University of London $752,660 Reactivation of MECP2 and CDKL5 genes by functional deactivation of Xist RNA $351,022 2017 TOTAL AWARDS $6,166,762 James Wilson, MD, PhD Katherin Meyer, PhD University of Pennsylvania Nationwide Children’s Hospital Gene therapy consortium Optimizing gene therapy for Rett Syndrome $1,585,886 $152,489 4 Katherin Meyer, PhD Clinical Trial Consortium Nationwide Children’s Hospital David Lieberman, MD, PhD A gene therapy consortium to develop and evaluate gene therapy Boston Children’s Hospital approaches in Rett Syndrome $395,000 $68,515 Clinical Trial Consortium Stuart Cobb, PhD Eric Marsh, MD, PhD University of Glasgow Children’s Hospital of Philadelphia Additional support for RNA-trans splicing efforts in Rett Syndrome $487,715 $290,000 Clinical Trial Consortium Rudolf Jaenisch, MD Alan Percy, MD, PhD Whitehead Institute for Biomedical Research University of Alabama Birmingham Reactivation of MECP2 with epigenome editing tools to rescue Rett Syndrome $495,000 $599,850 Clinical Trial Consortium Benjamin Philpot, PhD Jeffrey Neul, MD, PhD University of North Carolina Chapel Hill Vanderbilt University Medical Center Pilot study for reactivation of silenced MECP2 by artificial transcription factors $495,000 $145,443 Sasha Djukic, MD, PhD Q State Biosciences Albert Einstein College of Medicine Development of an in-vitro cell system for discovering and evaluating the effects Support for continuing work at the Rett Syndrome Center of therapeutic candidates on neurons produced using Rett patient iPS cells $103,000 $498,141 Huda Zoghbi, MD Michael Greenberg, PhD Baylor College of Medicine Harvard University Investigating the potential of antisense oligonucleotide therapy Development of an in-vitro cell system for discovering and evaluating the effects for MECP2 Duplication Syndrome of therapeutic candidates on neurons produced using Rett patient iPS cells $299,897 $55,826 Clinical Trial Consortium Daniel Tarquinio, DO Center for Rare Neurological Diseases $495,000 2016 TOTAL AWARDS $7,571,438 Adrian Bird, PhD / Michael Greenberg, PhD / Gail Mandel, PhD Stuart Cobb, PhD University of Edinburgh / Harvard University / Oregon Health and University of Glasgow Sciences University Scientific support for gene therapy, splicing therapy and protein MECP2 Consortium therapy programmes in Rett Syndrome $3,454,921 $210,000 Stuart Cobb, PhD / Steve Gray, PhD / Brian Kaspar, PhD Stuart Cobb, PhD / Gail Mandel, PhD / Alysson Muotri, PhD University of Glasgow University of Glasgow / University of North Carolina Chapel Hill / Optimizing MECP2 trans-splicing for human translation Nationwide Children’s Hospital / Oregon Health and Science University $330,804 / University of California San Diego Alysson Muotri A gene therapy consortium to develop and evaluate gene therapy University of California San Diego approaches in Rett Syndrome A drug-screening platform using MECP2-deficient human neurons $1,450,275 and preclinical testing $1,001,000 5 Alysson Muotri Michael Greenberg, PhD University of California San Diego Harvard University Role of an autism-related cytokine in a genetic model of ASD Identifying therapeutics for treating Rett Syndrome using nuclear (Autism Science Foundation) size as a proxy for long gene mis-regulation $12,500 $110,000 David Katz Q State Biosciences Case Western Reserve University School of Medicine Development of an in-vitro cell system for discovering and evaluating Preclinical studies of LM22A-4 in mouse models of Rett Syndrome the effects of therapeutic candidates on neurons produced using Rett $250,000 patient iPS cells $330,000 ArmaGen, Inc. Protein replacement for Rett Syndrome Miscellaneous Pilot Projects $33,838 $125,000 Rudolf Jaenisch, MD Sasha Djukic, MD, PhD Albert Einstein College of