Vertex, CRISPR's Gene Editing Drug Shows Positive Early Data

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) and partner CRISPR Therapeutics (NASDAQ:CRSP) announced positive preliminary safety and efficacy data from the ongoing phase I/II studies evaluating their CRISPR/Cas9 gene-editing therapy CTX001 for two severe blood disorders, namely sickle cell disease (SCD) and adult transfusion-dependent b-thalassemia (TDT)

The data was from the first two patients infused with CTX001 in the study, one from each disease. The patient with SCD was free of vaso-occlusive crises or pain crises, a painful complication of SCD disease, at four months following infusion versus 7 annualized events prior to therapy. The patient with TDT was transfusion independent at nine months after infusion versus 16.5 annualized transfusion rate prior to therapy.

Meanwhile, both patients achieved normal levels of haemoglobin and fetal hemoglobin on CTX001 infusion. While the TDT patient had total hemoglobin levels of 11.9 g/dL and 10.1 g/dL fetal hemoglobin, the same came in at 11.3 g/dL and 46.6%, respectively, for the patient suffering SCD.

Vertex believes that the interim data demonstrated meaningful benefits from CTX001 for patients following a one-time intervention.

Shares of Vertex and CRISPR Therapeutics were each up 2.5% and 17% on Tuesday in response to this news. In fact, this year so far, the CRISPR Therapeutics stock has skyrocketed 139.6% while Vertex has rallied 29.8% compared with the industry’s increase of 2.5%.

We remind investors that Vertex and CRISPR Therapeutics are co-developing CTX001, which is developed using CRISPR Therapeutics’ proprietary CRISPR/Cas9 technology for SCD and TDT in two separate phase I/II studies. Per the deal, the companies equally share all R&D costs and profits worldwide. Meanwhile, Vertex has rights to license up to six new gene editing treatments (including CTX001) developed using the CRISPR/Cas9 technology that emerged from the joint research deal.

In June 2019, Vertex announced the expansion of its collaboration with CRISPR Therapeutics to boost its gene editing capabilities for developing novel therapies to treat Duchenne muscular dystrophy (DMD) and Myotonic dystrophy type 1 (DM1).

Genomic editing technology using CRISPR technology to repair defective genetic material that causes diseases is probably one of the most promising and exciting healthcare innovations seen in decades. Though there are several methods to use CRISPR for targeting genetic defects that cause specific diseases, the most promising is the use of an enzyme called Cas9 to deliver CRISPR for the affected cells.

Apart from CRISPR Therapeutics, Intellia Therapeutics (NASDAQ:NTLA) and Editas Medicine, Inc (NASDAQ:EDIT) have candidates in pipeline, which are being developed using CRISPR/Cas9 technology to cure diseases. Intellia plans to submit an investigational new drug (IND) application for its CRISPR/Cas9 therapy NTLA-2001 in mid-2020 to treat transthyretin amyloidosis (ATTR). Meanwhile, Editas Medicine and its partner Allergan (NYSE:AGN) are conducting the Brilliance phase I/II study on EDIT-101 as a treatment option for Leber congenital amaurosis 10 (LCA10).

While CRISPR Therapeutics currently carries a Zacks Rank #3 (Hold), Vertex sports a Zacks Rank #1 (Strong Buy).You can see the complete list of today’s Zacks #1 Rank stocks here.

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