P.J. Brooks’ Post

Rare Diseases Research Innovation, NCATS, NIH at National Center for Advancing Translational Sciences (NCATS), NIH

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Professional Speaker and Patient Advocate at Self Employed

Today is very special! Today makes 5 years that I have been freed from Sickle Cell Disease! On July 2, 2019 I received my "Supacells"! God answered a childhood prayer and honored my leap of faith! The joy I have and the relief I feel can not be put into words! My life has been transformed Crispr technology. This is not an ad I just promised God that I would let the whole world know what He has done for me.I can not thank God enough! There is hope for everyone, no matter how grim the situation may be! I want to say thank you to my Sarah Cannon Institute/Tri-Star Centennial Hospital team, Misty, Katie, Dr. Domm and Dr. Frangoul They are all the angels that cared for me when I was at my lowest. I love you guys!#Miracle #Godisgood #CRISPR

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P.J. Brooks

Rare Diseases Research Innovation, NCATS, NIH at National Center for Advancing Translational Sciences (NCATS), NIH
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"CRISPR as a standard of medical care" for the treatment of monogenic #rarediseases sounds great to me

Fyodor Urnov

Scientific Director at Innovative Genomics Institute / Professor, MCB Department, UC Berkeley / Scientific co-founder, Tune Therapeutics
1w

"CRISPR as a standard of medical care" - this is the Innovative Genomics Institute mission as set by our founder, Jennifer Doudna. Melinda Kliegman, Ph.D., our Director of Public Impact, led an extraordinary KOL task force to develop actionable solutions to the key challenge of affordability. https://lnkd.in/g_vFq_RW

A roadmap for affordable genetic medicines - Nature

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P.J. Brooks

Rare Diseases Research Innovation, NCATS, NIH at National Center for Advancing Translational Sciences (NCATS), NIH
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Important update regarding the due date for applications to the Rare Disease Clinical Research Network #RDCRN #rarediseases https://lnkd.in/eHZVYiiV

Notice of Change for PAR-24-206 Rare Diseases Clinical Research Consortia (RDCRC) for the Rare Diseases Clinical Research Network (RDCRN) (U54 Clinical Trial Optional)

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P.J. Brooks

Rare Diseases Research Innovation, NCATS, NIH at National Center for Advancing Translational Sciences (NCATS), NIH
1w
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this is a really important step forward to LSDs, and more broadly for other potential accelerated approvals for #rarediseases based on #biomarkers
Denali Therapeutics

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1w

We are pleased to share the recent article titled, "Community Consensus for Heparan Sulfate as a Biomarker to Support Accelerated Approval in Neuronopathic Mucopolysaccharidoses" in Molecular Genetics and Metabolism. This article highlights the 2020 FDA guidance supporting accelerated approval based on substrate reduction for diseases like neuronopathic MPS, characterized by heparan sulfate (HS) in the brain. The article reflects the incredibly collaborative efforts across academia, advocacy, and industry, which culminated in the Reagan-Udall Foundation for the FDA-hosted workshop in February 2024. Appreciation to the authors and countless others for their unwavering commitment to advocating for science-driven approaches to accelerate the approval path for new treatment options for individuals and families living with MPS diseases. Read the full article here: https://lnkd.in/dNA49ThV Congratulations to the authors of the article: Joseph Muenzer, UNC at Chapel Hill; Carole Ho, Denali; Heather Lau MD MS, Ultragenyx; Mark Dant, The Ryan Foundation; Maria Fuller, University of Adelaide; Nidal Boulos, PhD, CCRP, Regenxbio; Patti Dickson, Washington University School of Medicine; Matthew Ellinwood, National MPS Society; simon jones, Manchester University NHS Foundation Trust; Eric Zanelli, Allievex Corp.; Cara O'Neill, Cure Sanfilippo Foundation. #MPS #defeatdegeneration
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P.J. Brooks

Rare Diseases Research Innovation, NCATS, NIH at National Center for Advancing Translational Sciences (NCATS), NIH
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great news and very much needed for #rarediseases https://lnkd.in/ekWcQj8B

FDA Rare Disease Innovation Hub to Advance Outcomes for Patients

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P.J. Brooks

Rare Diseases Research Innovation, NCATS, NIH at National Center for Advancing Translational Sciences (NCATS), NIH
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Always fascinating to see compelling data that undermines well-established scientific dogma. Here is another example relevant to endogenous DNA damage https://lnkd.in/eiSFcjwN

Mitochondrial H2O2 release does not directly cause damage to chromosomal DNA - Nature Communications

nature.com

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P.J. Brooks

Rare Diseases Research Innovation, NCATS, NIH at National Center for Advancing Translational Sciences (NCATS), NIH
4w Edited
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Are you interested in #geneediting ? Wouldn’t it be great to be able to detect on and off target edits in vivo in a non-invasive manner (e.g via lquid biopsies)?. If you think you can do this, or have other ideas for assays that would benefit clinical implementation of somatic gene edits, take a good look at this funding opportunity

Part 1. Overview Information

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