With the launch of its first near-curative gene therapy for sickle cell disease (SCD), everyone is talking about it CRISPR therapeutics (NASDAQ:CRSP) and its collaboration partner Vertex Pharmaceuticals.
But CRISPR isn’t the only biotech interested in advanced medicines, and investors are already looking for the next players who could make big breakthroughs. It’s riskier to invest earlier in a company’s development, but that’s precisely where the biggest returns lie, meaning it’s time to look to tomorrow’s rising stars.
While it’s unclear which companies will succeed or fail, there are a few early-stage gene editing companies worth keeping in mind. If their plans come to fruition – and that’s a big if – they could generate significant profits for their shareholders.
1. Intellia Therapeutics
Intellia Therapeutic (NASDAQ: EXTERNAL) could be the next CRISPR Therapeutics, as it is in clinical trials to develop curative gene-editing interventions for two inherited diseases: transthyretin amyloidosis (ATTR) and hereditary angioedema (HAE).
Its ATTR program is now entering Phase 3 trials, and the HAE program is completing enrollment for its Phase 2 study. If all goes as planned, it could also launch a Phase 1 trial in lung disease associated with carbon dioxide deficiency. alpha-1 antitrypsin (AATD) in 2024. This means that by 2030, it is possible (although unlikely) that the company could have three different genes. edition of drugs on the market.
It is important to note that Intellia is supported by Regeneron, which will be responsible for 25% of the costs and benefits of the ATTR program. This bodes well for its chances of becoming another CRISPR Therapeutics. Regeneron itself is about the same size as Vertex, which was instrumental in launching the first CRISPR program, so the company can bring substantial financial resources to bear to help Intellia if necessary.
So far, biotech doesn’t seem to need any help. Its cash and investments are worth $855 million, but its 12-month amount research and development (R&D) spending amounts to only $426 million.
But investors should be aware that it could face competition for market share in the ATTR market, assuming its treatment proves safe and effective and regulators agree to let the company have it. sell. As of December 21, AstraZeneca has just brought to market a therapy that treats polyneuropathy in the context of ATTR, so patients can avoid Intellia’s curative treatment.
But CRISPR Therapeutics also faces competition in its first market, so don’t count that business aside.
2. Therapeutic Verve
Therapeutic Verve (NASDAQ:VERV) has only one program in clinical trials, its candidate VERVE-101 to treat or cure heterozygous familial hypercholesterolemia (HeFH), which is in phase 1. In its initial form, VERVE-101 aims to permanently correct the problems of the genome of patients leading them to have dangerously high LDL-C cholesterol levels. But management believes that once there is proof of concept in the most severe patients, it may be possible to expand the scope of the project to eventually treat the 20% of the entire population who are at risk of developing atherosclerotic cardiovascular disease (ASCVD). . If this happens, it could ultimately treat hundreds of millions of people.
For now, Verve has a lot of work to do and has the right players on board to make serious progress. Its champion and collaborator is none other than Elie Lilly, a company with enormous resources. He also works with Vertex Pharmaceuticals.
Eli Lilly clearly sees promise in what biotech is working on. In late October, it purchased part of the marketing rights to VERVE-101 and a few other programs that Verve had initially licensed to another collaborator, Beam therapy, probably to ensure he could get in on the action. If the two men intensify or modify their collaboration again, it will be another bullish sign.
Verve’s chances of becoming the next CRISPR Therapeutics are also bolstered by its substantial third-quarter cash reserves of $485 million. It further raised $144 million in a public stock offering in early December, as well as $23 million in a private placement. Considering it only spent $154 million in cash over the last 12 months, it won’t even need to rely on its people anytime soon.
And that kind of strong track record means it’s a biotech stock worth watching, to say the least.
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Alex Carchidi has no position in any of the stocks mentioned. The Motley Fool holds positions and recommends Beam Therapeutics, CRISPR Therapeutics, Intellia Therapeutics and Vertex Pharmaceuticals. The Motley Fool recommends AstraZeneca Plc. The Mad Motley has a disclosure policy.
2 gene-editing stocks that could be the next CRISPR therapies was originally published by The Motley Fool