How does CRISPR, the gene editing inventor, make money?

CRISPR

Please see my post of “World’s first gene editing therapy Casgevy finally approved” as affilated article.

Pioneer of gene editing

CRISPR Therapeutics (ticker: CRSP) is a biotech company headquartered in Switzerland. It is one of the first companies to use the CRISPR gene-editing platform to develop drugs for a variety of rare and common diseases.

Nobel Prize Winner in Chemistry

CRISPR was founded in 2013 by Emmanuelle Charpentier, Shaun Foy and Rodger Novak. [6] Charpentier later shared the 2020 Nobel Prize in Chemistry with Jennifer Doudna. As a member of the working group, she provided the first scientific documentation on the development and use of CRISPR gene editing. This allows DNA to be specifically modified and edited so that it can be used to ameliorate disease. CRISPR is applying this technology platform to research, develop and commercialize drugs to treat a variety of diseases, including sickle cell disease, beta thalassemia, various cancers, type 1 diabetes and cardiovascular disease.

Main products

CRISPR Therapeutics is developing a variety of drugs to treat blood disorders, cancer, diabetes and other serious diseases.

CRISPR remains on track to start clinical trials for CTX112, a gene-editing treatment for systemic lupus erythematosus. In 2024, in addition to ongoing cancer and autoimmune disorder trials.

Cooperate with Vertex

Since CRISPR is an emerging small biotechnology industry, in order to survive and expand its business, it is no different from other new biotechnology entrepreneurs. It must first find a cooperation with a large international pharmaceutical company to achieve the company’s business goals.

As a result, Vertex and CRISPR entered into a strategic research collaboration in 2015 focused on using CRISPR/Cas9 to discover and develop potential new therapies targeting the underlying genetic causes of human disease.

Main competitor

Bluebird Bio

The FDA approved Bluebird Biotechnology (ticker: BLUE)’s Lyfgenia for sickle cell disease in December, 2023, along with a rival gene therapy for the disease developed by Vertex Pharmaceuticals and CRISPR Therapeutics.

It has gene-editing treatments for TDT and SCD on the market. But there are three reasons why it should not be a big threat:

  • First, Bluebird Biotechnology only operates in the United States.
  • Second, Vertex is a much larger biotech company than either Bluebird Biotech or CRISPR, and has a track record of negotiating lucrative deals with third-party payers.
  • Third, Bluebird Biotech’s SCD treatment Lyfgenia carries a boxed warning for blood cancers. In the end, Lyfgenia cost $3.1 million, while Casgevy cost $2.2 million.

Latest quarterly result

Announced “profitable” results for the first quarter of 2024 looked more like losses, causing the company to close down 8.5%. CRISPR’s revenue fell 99.5% year-over-year due to the lack of major milestone payments like those in the first quarter of 2023, and the company reported a loss of $1.43 per share, more than double its loss in the same period last year.

Still unprofitable

With only $166.5 million in quarterly negative free cash flow in Q1 2024 report.

However, CRISPR has at least three years before it’s likely to need additional cash. This is because the company’s Casgevy treatment for sickle cell anemia, developed in partnership with Vertex Pharmaceuticals has been approved by FDA. It could rely on 40-60 revenue split on sales between CRISPR and Vertex.

Capital market performance

CRISPR’s partner Vertex Pharmaceuticals’ stock price rose by approximately 42.26% in one year in 2023; during the same period, CRISPR Therapeutics rose by 52.68%.

CRISPR
credit: Wiki

I am the author of the original text, the essence of this story was originally featured on Smart Magazine, Issue of October 2024

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