Three ‘Strong Buy’ Biotechs To Tap Now

If you haven’t heard of CRISPR technology before, listen up. This potentially revolutionary technology enables scientists to essentially fix faulty genes. Such technology has been described as a medical miracle with transformative effects for countless diseases from blood diseases, genetic deafness, genetic blindness to neurological disorders.

However, to achieve this goal, companies have to fulfill multiple complex requirements. This includes: efficiently editing a wide range of mutations, reaching the site of the disease, precisely cutting the DNA, and achieving the right to repair. So which biotechs are paving the way?

Vertex Pharmaceuticals (VRTX)

For exposure to the CRISPR space without the risk, it’s worth checking out large-cap biotech Vertex. Symdeko, Vertex’s new-and-improved cystic fibrosis combo, is the company’s big money generator. But Vertex has also partnered with small-cap CRISPR Therapeutics (covered below) in the development of the promising CTX001 gene-editing therapy for β Thalassemia and Sickle Cell Disease. Given that this is a market of 360,000 births/ year, and the treatment could cost $450,000-$700,000 per patient, this could be a serious money-maker for both companies.

Currently CRSP and VRTX are splitting the R&D costs involved to develop this drug. The four-year deal involves Vertex paying CRSP $105 million—$75 million in cash, and $30 million via an equity investment. So where are we now? According to the company’s recent presentation, it expects to initiate Phase 1/2 clinical studies of CTX001 this year. This is from CRSP’s website:

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“As a company founded on innovative science, we’re excited to begin this collaboration with CRISPR, as it puts us at the forefront of what we believe may be a fundamental change in the future treatment of disease—using gene editing technologies to address the underlying genetic causes of many diseases,” Vertex’s CSO David Altshuler said in a statement back in 2015 when the deal began.

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