Research programme: genome editing therapeutics - Editas Medicine

Drug Profile

Research programme: genome editing therapeutics - Editas Medicine

Alternative Names: LCA10

Latest Information Update: 02 Feb 2017

Price : $50

At a glance

  • Originator Editas Medicine
  • Class Gene therapies
  • Mechanism of Action Gene modulators
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Alpha 1-antitrypsin deficiency; Cystic fibrosis; Duchenne muscular dystrophy; Eye disorders; Haematological disorders; Leber congenital amaurosis

Most Recent Events

  • 09 Jan 2017 Editas announces intention to submit IND application to US FDA for the LCA 10 programme by end of 2017
  • 09 Aug 2016 Editas Medicine and Adverum Biotechnologies collaborate to evaluate genome editing therapeutics with AAV vectors for Eye disorders
  • 28 Jul 2016 Editas Medicine and Fondazione Telethon and Ospedale San Raffaele agree to co-develop genome edited hematopoietic stem cell (HSC)
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