First Human CRISPR Trials By Editas Gets FDA Approval
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First Human CRISPR Trials By Editas Gets FDA Approval

Editas is back in the race after a prolonged absence. In a revolutionary decision, FDA has approved Editas IND application for its LCA10 (Leber Congenital Amaurosis type 10 ) candidate EDIT-101 which will enable them for human testing of its CRISPR-based treatment for LCA10, a rare form of blindness. It will start enrolling approx 20 patients carrying IVS26 mutation in a phase 1/2 trial and is slated to become the first in vivo CRISPR genome editing treatment ever. 

As a consequence of the IND approval, Allergen will fund Editas with $25 million milestone payment. Under a $40 million agreement made earlier, Allergan agreed to develop and commercialize EDIT-101.

Editas registered in October for the IND. Earlier this month, Editas revealed the NIH decided that a Recombinant DNA Advisory Committee review of the protocol for the Stage I/II trial of EDIT-101 wasn’t necessary.

“The FDA’s acceptance of our IND for EDIT-101 is a significant moment in the field of genome editing, and importantly, a critical milestone for patients, as we are now one step closer to a treatment for LCA10,” Katrine Bosley, Editas’ president, and CEO, said in a statement.

Editas was earlier expected to

submit EDIT-101 for approval by end of 2017, but owing to some manufacturing difficulties the process got delayed. Meanwhile, companies like CRISPR Therapeutics and partner Vertex took the opportunity to march ahead in the race. An ex-vivo CRISPR-based treatment for beta-thalassemia was initiated earlier this year but upon FDA’s orders, clinical trails were kept on hold which was lifted off ahead in the month of October.

LCA10, the most common cause of inherited childhood blindness is caused by a mutation in the CEP290 gene. By the use of  EDIT-101, Editas is aiming to eliminate the mutation using CRISPR. In the process, it will remove the mutated nucleotide and surrounding DNA which will enable restoration of the normal protein expression and function of the remaining photoreceptor cells, arresting the further loss of vision for patients with LCA10.

As per a R&D deal signed in between Editas and Allergan last year in March, Editas received an upfront $90 million from Allergen in return of which Allergan gained exclusive access and licensing option to up to five of Editas’s early-stage CRISPR genome-editing programs targeting eye diseases, including EDIT-101.

Editas, the first CRISPR based gene editing research company was founded in November 2013 and bagged initial funding from $43 million from Third Rock Ventures, Polaris Ventures, and Flagship Ventures. Later Editas was able to pool in up to $120 Million in funding from Bill Gates along with 13 Other Investors including Google Ventures.

Recently an announcement by a Chinese scientist who claims to create worlds first CRISPR edited babies has taken the scientific community by shock. But no proper documented evidence and research results have been provided yet which makes the claims by the Chinese scientist He Jiankui hard to believe. Investigations are still under process to validate its authenticity.

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