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​01/07/21


ADNP, One of 3 Genes Selected for
Mount Sinai & Rumi Scientific
Partnership to Advance Drug Discovery for Rare Genetic Disorders Tied to Autism 


Collaboration to produce human neural cell organoids and a platform for mid-throughput drug screenings.
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​
​Cranes New York announced today that Mount Sinai's Icahn School of Medicine on Wednesday announced a partnership with Rumi Scientific, a drug discovery company based in Midtown, to research rare genetic disorders that carry a high risk of autism.


“The collaboration is to produce human neural cell organoids in lab dishes and see if we can develop a platform for mid-throughput drug screening and ADNP was one of 3 genes selected”, said Joseph Buxbaum, director of the Seaver Autism Center and professor of psychiatry, neuroscience, and genetics and genomic sciences at Icahn School of Medicine to Sandra Bedrosian-Sermone, President of the ADNP Kids Research Foundation.

“We have focused on three genes that are involved in development, since the assay reflects early brain development”, said Buxbaum.

“ADNP is repeatedly identified as one of the top genes for autism and developmental delay”, said Bedrosian-Sermone, and continued “we can’t be more thankful to both Mount Sinai and Rumi Scientific for selecting ADNP in this potentially life changing study that could help find treatments for individuals with ADNP Syndrome.”

Icahn's Seaver Autism Center for Research and Treatment will provide patient-derived stem cells as well as engineered ones to Rumi, which uses a technology platform conceived at Rockefeller University to create tissue cultures to express specific genetic mutations. The cultures provide understanding into how gene mutation leads to autism and the potential for finding drugs to treat it.

"It's time to start thinking about using precision medicine in developmental disorders like autism," said Buxbaum.

The first portion of the partnership—growing out the cultures—should conclude by the middle of the year or even sooner, said Ilona Nemeth, CEO of Rumi Scientific. It is hoped assays for drug discovery could be ready by next year, Buxbaum said.

The two organizations began conversations about the partnership in early 2020, and Mount Sinai is Rumi's first academic institution partnership, Nemeth said.

Mount Sinai and Rumi are putting financial resources into this partnership, Buxbaum and Nemeth said, but both declined to provide details.

Much neurodegenerative brain disease research fails to enter into trials due to a lack of representative human models, Nemeth said. "This partnership allows us to achieve representative human tissue, and Seavers has those genes. This could allow us to succeed where many others have failed," she said.

Rumi Scientific was founded in 2016 and is funded by government grants, customer revenue and anchor investor funds. It received about $3 million in grants, and its revenue and raised capital are undisclosed. —S.S.

​The story was picked up by Crains New York
​
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Disclosure: This page represents information known to the best of our knowledge.  Given the sensitivity and fast changing pace of research and drug development, some of the information posted on our website www.adnpfoundation.org may be inaccurate or not complete, therefor this information should be confirmed by the reader. Additionally, the medical information on this website is not substitute for personal diagnosis or medical care advise. Families and patients should consult a medically qualified clinician in all matters relating to genetic diagnosis, disease management and health. Information on ADNP research is a very fast-moving field and while the information here is believed to be the best available at the time of publication, some facts may later be updated or change. Please see additional disclosures on our PRIVACY POLICY page.
  • About
    • What is ADNP
    • Our Story
    • Our Mission
    • Our Team
    • Medical and Scientific Advisory Board
    • 501(c)(3) Determination
    • Inclusivity Statement
  • RESEARCH
    • Research Projects
    • Research Strategy
    • Ketamine Trial
    • Ketamine Trial Publication
    • Ketamine Update
    • Path to a cure
    • NET STUDY
    • publications
  • Families
    • Newly Diagnosed
    • ADNP Patient Registry
    • Contact Registry
    • Understanding the Basics
    • Worldwide Parent Ambassadors
    • contact us
    • ADNP Family Conference >
      • HIGHLIGHTS- ADNP Syndrome Family Conference
      • TSA Travel Help
      • #CureADNP
  • NEWS
    • NET Collaboration Study
    • Ketamine Trial Phase 2 Announcement
    • Mount Sinai Launches First Drug Trial for ADNP Syndrome
    • Seaver Midtown Partnership
    • Biomarker Discovered
    • NEWS - p.Tyr719* Case Study
    • NEWS HIGHLIGHTS- ADNP Syndrome Family Conference
  • ADNP STORE
  • DONATE/SUPPORT
    • Warrior Fun Run 2022 >
      • Past Fun Run Pictures
      • Sponsorship Opportunity
    • Boston Marathon
    • Third Party Fundraising
  • About Us new