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CRISPR (CRSP) & Vertex Complete BLA Submission for Exa-Cel

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CRISPR Therapeutics (CRSP - Free Report)  and Vertex Pharmaceuticals (VRTX - Free Report) completed the rolling submission of biologics license applications (BLAs) to the FDA for exagamglogene autotemcel (exa-cel) to treat sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). The companies started the BLA submission in November 2022.

Exa-cel is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy, developed to treat SCD or TDT. The completion of the BLA filing for exa-cel is a milestone as it is the first regulatory filing for a CRISPR-based treatment within a decade of the CRISPR platform’s discovery.

The BLA submission is supported by data from the phase III studies — CLIMB-111, CLIMB-121 and CLIMB-131, a long-term follow-up study.

The BLAs also include a request for priority review, which, if granted, would shorten the FDA's review timeline to eight months from a standard 12 months.

Shares of CRISPR have plunged 32.9% in the past year compared with the industry's 15.2% decline.


Zacks Investment Research
Image Source: Zacks Investment Research


Shares of Vertex have gained 17.2% in the past year against the industry's 15.2% decline.


Zacks Investment Research
Image Source: Zacks Investment Research


Vertex and CRISPR also filed similar submissions on exa-cel in Europe and the U.K. in December 2022 that were validated by the European Medicines Agency (EMA), and the Medicines and Healthcare products Regulatory Agency in January 2023.

In the United States, exa-cel has been granted Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations from the FDA for both SCD and TDT. In the EU, the same has been granted Orphan Drug Designation and Priority Medicines designation from the European Commission and the EMA, respectively, for both SCD and TDT.

CRISPR Therapeutics is also developing two gene-edited allogeneic cell therapy programs, chimeric antigen receptor T cell (CAR-T) candidates, CTX110 and CTX130, for hematological and solid tumor cancers.

Vertex has a portfolio of marketed products to treat cystic fibrosis. These are Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), Symdeko/Symkevi (tezacaftor/ivacaftor and ivacaftor), Orkambi (lumacaftor/ivacaftor) and Kalydeco (ivacaftor).

The company is also developing treatments for neuropathic pain, APOL1-mediated kidney disease, type I diabetes and alpha-1 antitrypsin deficiency. It also has earlier-stage programs in conditions like muscular dystrophies.

Zacks Rank & Another Stock to Consider

Currently, CRISPR has a Zacks Rank #2 (Buy).

Another top-ranked stock in the same sector is Kala Pharmaceuticals (KALA - Free Report) . Kala Pharmaceuticals currently carries a Zacks Rank #2. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here.

Loss per share estimates for KALA have narrowed from $19.67 to $15.35 for 2023 and from $14.41 to $13.12 for 2024 in the past 60 days. The company’s shares have plunged 76.2% in the past year.

KALA’s earnings beat estimates in two of the last four quarters and missed the mark in the other two, the average surprise being 11.56%.

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