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Emerging from Stealth Mode, Gene Editing Startup Prime Medicine Raises $315M


Emerging from Stealth Mode, Gene Editing Startup Prime Medicine Raises $315M
July 14, 2021
Source: CIPhotos/Getty Images
Prime Medicine, whose “search and replace” approach to gene editing underpins the second genome editing technology to be commercialized by the lab of David R. Liu, Ph.D., and colleagues, said today it has completed $315 million in financing, ending nearly a year of operation in stealth mode.
The financing consists of a $115 million Series A round and a $200 million series B round. Prime said proceeds from the financing will be used to continue building the company, rapidly advance towards clinical indications for its gene editing therapy candidates, and expand the capabilities of its platform. ....

Davidr Liu , Keith Joung , Andrew Anzalone , Keith Gottesdiener , Feng Zhang , Regeneron Pharmaceuticals , Broad Institute , Merkin Institute Of Transformative Technologies In Healthcare , T Rowe Price Associates Inc , Harvard University , Redmile Group , Cold Spring Harbor Laboratory , Prime Medicine , Intellia Therapeutics , New England Journal , Richard Merkin Professor , Merkin Institute , Transformative Technologies , Cold Spring Harbor , Prime Editing , Beam Therapeutics , F Prime Capital , Casdin Capital , Cormorant Asset Management , Moore Strategic Ventures , Public Sector Pension Investment Board ,

Genetic base editing treats sickle cell disease in mice


Broad Institute of MIT and Harvard
Sickle cell disease (SCD) is the most common deadly genetic disorder, affecting more than 300,000 newborns worldwide each year. It leads to chronic pain, organ failure, and early death in patients. A team led by researchers at the Broad Institute of MIT and Harvard and St. Jude Children’s Research Hospital has now demonstrated a base editing approach that efficiently corrects the mutation underlying SCD in patient blood stem cells and in mice. This gene editing treatment rescued the disease symptoms in animal models, enabling the long-lasting production of healthy blood cells.
The root of SCD is two mutated copies of the hemoglobin gene, HBB, which cause red blood cells to transform from a circular disc into a sickle shape setting off a chain of events leading to organ damage, recurrent pain, and early mortality. In this study, the researchers used a molecular technology called base editing to directly convert a single letter of patho ....

David Liu , Gregory Newby , Kaitly Woodard , Mitchell Weiss , Broad Institute , Harvard University , Merkin Institute Of Transformative Technologies In Healthcare , Jude Children Research Hospital , Jude Department Of Hematology , Howard Hughes Medical Institute , Jude Children , Research Hospital , Richard Merkin Professor , Merkin Institute , Transformative Technologies , Jude Department , Jonathan Yen , Thiyagaraj Mayuranathan , Cell Biology , Chronic Pain , Sickle Cell , Stem Cells , டேவிட் லியூ , கிரெகொரி புதியவர் , மிட்செல் வெயிஸ் , பரந்த நிறுவனம் ,

Breakthrough Study Supports Genetic Editing as a Potential Treatment for Rare Rapid-Aging Disease Progeria


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PEABODY, Mass., Jan. 6, 2021 /PRNewswire/
 The Progeria Research Foundation (PRF) announced today the publication of a breakthrough preclinical mouse study in the journal
Nature, demonstrating the substantial effect of genetic editing in directly reversing the mutation that causes Hutchinson-Gilford Progeria Syndrome (HGPS, or Progeria). Scientists at Broad Institute, the National Institutes of Health (NIH), PRF and others contributed to this ground-breaking study, indicating a critical new pathway toward correcting the root cause of Progeria.
Progeria is an ultra-rare, fatal, pediatric rapid-aging disease, affecting approximately 400 children worldwide. Without the FDA-approved treatment Zokinvy (lonafarnib), children with Progeria die of heart disease at an average age of 14.5 years, due to premature atherosclerosis resulting in heart attacks. Zokinvy increases average lifespan by 2.5 years and improves some symptoms of Progeria, but it do ....

United Kingdom , David Liu , Francis Collins , Sam Berns , Leslie Gordon , Jonathan Brown , Division Of Cardiovascular Medicine At Vanderbilt University , Broad Institute , Merkin Institute Of Transformative Technologies In Healthcare , National Human Genome Research Institute , International Progeria Patient Registry , Progeria Research Foundation , Richard Merkin Professor , Merkin Institute , Transformative Technologies , Senior Investigator , Assistant Professor , Cardiovascular Medicine , Progeria Patient Registry , ஒன்றுபட்டது கிஂக்டம் , டேவிட் லியூ , பிரான்சிஸ் கோலின்ஸ் , சாம் பெர்ன்ஸ் , லெஸ்லி கோர்டந் , ஜொனாதன் பழுப்பு , பிரிவு ஆஃப் இருதய மருந்து இல் வாண்டர்பில்ட் பல்கலைக்கழகம் ,