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Biotech / Medical : Biotech Valuation
CRSP 56.38-9.0%Nov 4 3:59 PM EST
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To: JamesK who wrote (52044)3/27/2023 12:08:58 PM
From: Art Bechhoefer1 Recommendation

Recommended By
OldAIMGuy

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The research paper published in the March 17 issue of Science provided details on the underlying concepts for understanding how diseases like ALS, frontal temporal dementia, and Alzheimer's progress. Ionis Pharma uses its antisense technology to create a molecule that binds to an unwanted protein located at the wrong site in order to prevent further cell proliferation and progression of the disease.

Ionis has at least three separate development stage drugs incorporating these concepts. Two of them (a type of ALS and an Alzheimer's drug that stops the proliferation of tau tangles, are in phase 2 or phase 3 clinical trials. Ionis is the leading developer of antisense technology and owns key patents on the technology as well as the specific drugs that use the technology. Antisense is the same technology used for Spinraza, the most successful treatment for spinal muscular atrophy, which now generates about $1 billion sales annually, with profits split between Ionis and its collaborating partner Biogen. The same process for creating the molecule that stops cell proliferation in spinal muscular atrophy is used to create other antisense molecules, which by their nature act so specifically (on just one type of cell) that they tend to have fewer side effects than other competing treatments.

Art
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