Intellia Therapeutics (NTLA) used CRISPR gene editing to reduce the problem protein in patients, but NTLA shares withdrew on Tuesday over patent and competition issues.
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Treatment with the CRISPR gene editing company reduces the protein transthyretin in patients with a disease that causes it to build up on nerves. This leads to systemic problems. The results lasted from 2 to 12 months in 15 patients and proved to be relatively safe.
But the results came when the U.S. Patent and Trademark Office said a team led by Feng Zhang of the Broad Institute had actually invented the technology. The institute has exclusively licensed its technology for Editas Medicine (EDITING). Intellia uses patents held by the University of California, the University of Vienna and CRISPR pioneer Emmanuel Charpentier, also known as CVC. Charpentier and Jennifer Dudna won the 2020 Nobel Prize in Chemistry for their work at CRISPR.
“Intellia is carefully reviewing the decision and we are confident that CVC will find a way forward to confirm its rights (intellectual property), including the possibility to appeal to the Federal Circuit,” an Intellia spokesman said in an email to Investor’s Business Daily.
In addition, analysts noted that Intellia’s results are not much better than Alnylam Pharmaceuticals“(ALNY) Onpattro. Onpattro treats patients with related heart disease.
Shares of NTLA fell 20.1% to 79 in lunchtime trading on the stock market today. Shares of Crispr Therapeutics (CRSP) also fell 5.8% to close to 57.80. Shares of Editas rose 5% to 18. Shares of Alnylam also rose 3.2% to 162.90. Shares of Intellia partner, Regeneron Pharmaceuticals (REGN) fell 1.8% to 607.30.
NTLA shares dives while racing sets
Intellia is testing four doses of its single drug. The best response comes from the highest dose, resulting in a 93% average reduction in transthyretin protein. Protein levels remained low during the follow-up period, which ranged from two to 12 months.
Patients who received lower doses showed an average reduction of 52% -87%.
But Alnylam’s Onpattro – which treats patients with the same disease that causes protein to build up in the heart – leads to an 89% reduction in protein at six months and an 84% reduction at 18 months.
(Of course, (Intellia medicine) has the advantage of being a potential single-dose treatment, but we see additional competition from Alnylam (the drug called vutrisiran), which demonstrates similar levels of (transthyretin) reduction and is dosed in a relatively convenient format for subcutaneous injection every three months, ”said Wedbush analyst David Nirengarten in a report to clients.
It has a neutral rating and a 108 price target for NTLA shares.
The results reinforce Intellia’s leadership with the systemic in vivo CRISPR gene editing drug. In vivo means that the editing of genes takes place inside the body. Systemic means that the drug enters intravenously and must find its way to the right organ – in this case the liver.
Crispr works on ex vivo treatment, which means that it edits cells outside the body and then infuses them into patients. Editas is an in vivo treatment, but patients receive treatment directly in the eye.
The CRISPR-based medicine seems safe
CRISPR gene editing treatment seems safe. Only one patient experienced a severe side effect – vomiting. This patient had a history of gastroparesis, a condition in which the stomach cannot be emptied properly.
At all four dose levels, the most common side effects are headache, infusion reactions, back pain, rash and nausea, said SVB Leerink analyst Manny Foruhar in a note to clients. The company has not reached the maximum tolerated dose, which means that it has the potential to deliver higher doses safely.
Foroohar kept its rating outperforming NTLA shares.
Follow Alison Gatlin on Twitter at @IBD_AGatlin.
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