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CRISPR (CRSP) Inks Deal for Gene Therapies in Neurological Diseases

·3 min read

CRISPR Therapeutics AG CRSP announced that it has entered into a strategic partnership with privately held biotech Capsida Biotherapeutics Inc. to develop and commercialize in vivo gene editing therapies for the treatment of familial amyotrophic lateral sclerosis (ALS) and Friedreich’s ataxia.

The collaboration will leverage Capsida’s AAV engineering platform with CRISPR Therapeutics’ gene-editing platform to generate transformative gene-edited therapies for various neurological conditions.

Per the deal, CRISPR Therapeutics will be leading the research and development (R&D) of the Friedreich’s ataxia program and provide gene-editing activities for both indications. Meanwhile, Capsida will carry out R&D of the ALS program and conduct capsid engineering for both programs.

Both CRISPR Therapeutics and Capsida will have the option to co-develop and co-commercialize the program that the other company leads, upon potential approval. Both companies will then equally share all the research, development and commercialization costs and profits globally, if a product is generated from this collaboration.

Shares of CRISPR Therapeutics have declined 19.2% so far this year against the industry’s increase of 0.9%.

Zacks Investment Research
Zacks Investment Research

Image Source: Zacks Investment Research


We note that CRISPR Therapeutics is actively seeking collaborations and leveraging its CRISPR/Cas9 gene-editing platform to make therapies for hemoglobinopathies, cancer, diabetes and other diseases.

Its lead pipeline candidate is CTX001, an investigational ex vivo CRISPR gene-edited therapy, which is being developed in mid-stage studies for treating transfusion-dependent beta thalassemia (TDT) and sickle cell disease (SCD).

CTX001 is being developed under a co-development and co-commercialization agreement with Vertex Pharmaceuticals VRTX. The companies entered into a strategic research collaboration in 2015 for developing CTX001.

CRISPR Therapeutics also inked a new option deal with Bayer BAYRY, which carries an option to co-develop and co-commercialize two products for the treatment of certain autoimmune disorders, eye disorders or hemophilia A disorders.

Zacks Rank & Stock to Consider

CRISPR Therapeutics currently carries a Zacks Rank #3 (Hold). A better-ranked stock in the biotech sector is Kamada Ltd. KMDA, which has a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Kamada’s earnings estimates have been revised 21.4% upward for 2021 and also moved 18.1% north for 2022 over the past 60 days.

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Vertex Pharmaceuticals Incorporated (VRTX) : Free Stock Analysis Report

Bayer Aktiengesellschaft (BAYRY) : Free Stock Analysis Report

Kamada Ltd. (KMDA) : Free Stock Analysis Report

CRISPR Therapeutics AG (CRSP) : Free Stock Analysis Report

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