Fragile X drug developer Fulcrum to use Horizon's CRISPR to find gene targets

By Gareth Macdonald contact

- Last updated on GMT

Fragile X drug developer Fulcrum to use Horizon's CRISPR to find gene targets

Related tags: Dna, Medicine, Therapy

Fulcrum Therapeutics will use a CRISPR gene editing-based platform to identify genetic targets under a deal with Horizon Discovery Group.

The deal – financial terms of which were not made public – will see Horizon apply its platform and extensive know-how to identify novel gene regulation targets for Fulcrum for the development drugs and therapies.

The project will run for at least the next five months and will initially focus on two specific rare diseases, although the deal allows for additional targets to be added.

Specifics of the research were not disclosed. Fulcrum’s website states that it is currently focused on developing therapies for Fragile X syndrome (FXS) and a rare type of muscular dystrophy called facioscapulohumeral muscular dystrophy (FSHD).

Robert Gould, Fulcrum Therapeutics’ CEO, said: "Horizon's reputation in gene editing and its applications, including CRISPR-based screening, is unparalleled. It was natural for Fulcrum to turn to Horizon as a long-term partner. 

With their deep scientific expertise, and with a broad IP portfolio underpinning their work, we are confident in a productive and exciting collaboration​."

Fulcrum was set up in July by Boston, US-based venture capital organisation Third Rock Ventures. The firm aims to discover and develop small molecule pharmaceuticals that target genes involved in a range of diseases.

Gene editing services

Horizon CEO, Darrin Disley, welcomed the Fulcrum deal and predicted that gene editing techniques like CRISPR will play an important role in therapeutic development.

CRISPR-based screening promises to be a powerful tool for the identification of novel targets, and the collaboration with Fulcrum demonstrates the potential for this technology to be used in areas outside of oncology​” he said.

Related topics: Preclinical Research, Preclinical

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