editas gene editing

Editas Gene-Editing Pipeline Will Treat Neurological Diseases

editas gene editing

Editas Medicine, Inc. (EDIT) makes medicines to treat serious diseases using its proprietary genome editing platform based on CRISPR technology. In an effort to explore the in vivo delivery of gene editing medicines to treat neurological diseases, the company recently announced its strategic research collaboration with Asklepios BioPharmaceutical (AskBio), a privately-held gene therapy company, leaving the financial terms undisclosed.

There is no doubt that the need for treatment of neurological diseases remains unmet and the collaboration between the two companies aims to integrate AAV technology and clinical-stage manufacturing with EDITAS gene editing technologies.

Editas Gene Editing: Working hard to find a cure

EDIT-101 is the company’s lead pipeline candidate, which is being developed in partnership with Allergan AGN. It uses CRISPR gene editing to treat Leber congenital amaurosis type 10 (LCA10) – a rare genetic illness that causes blindness. The two companies are planning to initiate patient dosing in phase I / II dose-escalation study called Brilliance on EDIT-101 for addressing LCA10 in the second half of 2019. The study opened for patient enrolment in July.

This year, shares of Editas went down 10.9% compared to the 6.4% decrease in the industry. 

Other than LCA10, EDIT is also in the pursuit of the development of CRISPR candidates for eye diseases, including Usher Syndrome type 2A (USH2A) and the recurrent ocular Herpes Simplex Virus type 1 (HSV-1).

EDIT is also in collaboration with big pharma entities for its CRISPR technology, providing research support and sufficient funds to fulfil its pipeline development plans. The company has a licensing pact with Juno Therapeutics which is now part of Celgene CELG. Using the latter’s gene-editing approaches including CRISPR-Cas9, both companies aim to develop the engineered T cell medicines to find a cure for cancer.

EDITAS also signed a research and cross-licensing deal with privately held BlueRock Therapeutics, LP, in April 2019. They aim to combine their respective genome editing and cell-therapy technologies to come up with engineered cell medicines.


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