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Editas' SCD Candidate Gets FDA's Rare Pediatric Disease Tag

Editas Medicine, Inc. EDIT announced that the FDA has granted Rare Pediatric Disease (RPD) designation to its experimental CRISPR medicine EDIT-301, which is being developed as a potential medicine for sickle cell disease. The company plans to file an investigational new drug application (IND) by the end of 2020 to begin clinical studies on EDIT-301 for the given indication.

Notably, the RPD tag is granted by the FDA in case of serious or life-threatening diseases, which usually affect less than 200,000 people in the United States, primarily individuals aged 18 years and below.

Additionally, the RPD status conferred on the candidate will provide Editas with an opportunity to obtain a Pediatric Disease Priority Review Voucher from the FDA after the regulatory body approves a qualifying new drug application (NDA) or a biologics license application (BLA) for the treatment of any rare pediatric disease. The company may use the voucher to obtain a Priority Review for an NDA or a BLA submitted later.

Shares of Editas have rallied 15.6% so far this year compared with the industry’s increase of 1.4%.


Editas is a development-stage genome editing company, which makes medicines to treat serious diseases using its proprietary genome editing platform based on CRISPR technology.

The company’s lead pipeline candidate is EDIT-101 that employs CRISPR gene editing to treat Leber congenital amaurosis type 10 (LCA10), a rare genetic illness that causes blindness. LCA10 disease has a significant unmet need as no therapies are presently approved to cure the disease either in the United States or in the EU.

In August 2020, Editas terminated its 2017 agreement with Allergan [now part of AbbVie ABBV] and regained full global rights to develop, manufacture and commercialize its ocular medicines including EDIT-101.

Earlier, the company with former partner Allergan initiated patient dosing during March 2020 in the phase I/II dose escalation study called Brilliance to evaluate EDIT-101 for LCA10. Editas plans to complete dosing of the adult low-dose cohort (two patients) and dose at least one patient in the adult mid-dose cohort by this year-end.

Zacks Rank & Stocks to Consider

Editas currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the healthcare sector include Emergent BioSolutions Inc. EBS and Horizon Therapeutics Public Limited Company HZNP, both sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

Emergent’s earnings estimates have moved 62.3% and 78.8% north for 2020 and 2021, respectively, over the past 60 days. The stock has skyrocketed 132.7% year to date.

Horizon’s earnings estimates have been revised 61.5% and 57.7% upward for 2020 and 2021 each over the past 60 days. The stock has rallied 102.5% year to date.

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