2/7/2024 0 Comments Tessera therapeutics stocksThis treatment is injected directly into the eye. But the Massachusetts biotech's most advanced program is a treatment for a rare genetic eye disorder called Leber congenital amaurosis. Like other companies, Editas is focused on treating sickle-cell disease and beta thalassemia by using CRISPR outside the body. What Editas is working on: Editas has also begun human testing for its first gene-editing programs, which feature a mix of editing inside and outside the body. "We are ever so close to a CRISPR-based commercial drug, which will be the first of its kind in the world," CRISPR Therapeutics CEO Samarth Kulkarni previously told Insider. The treatment is being developed in collaboration with the Boston biotech giant Vertex to treat sickle-cell disease and beta thalassemia. What to watch for: A key milestone for the gene-editing field could come this year, as CRISPR Therapeutics expects to submit the first CRISPR drug for regulatory approval before the year's end. It's working with a biotech called ViaCyte, and the two hope to start human testing this year. Latest news: In November, CRISPR's experimental diabetes treatment was cleared to start an initial human study in Canada. The biotech announced its first gene-editing results in humans in November 2019, publishing data from the first two patients treated for genetic blood disorders. What CRISPR Therapeutics is working on: CRISPR Therapeutics has several CRISPR-based therapies in clinical trials to treat certain types of cancer, type 1 diabetes, sickle-cell disease, and beta thalassemia. The first glimpse of data in June was a high point for the entire biotech industry - 2022 will see whether that promise holds up in more patients.ĬRISPR Therapeutics CEO Samarth Kulkarni. ![]() What to watch for: Because it's a company now firmly in the clinic, you can expect a lot more human data from Intellia in 2022, including updated results for the amyloidosis treatment and the first interim results for its hereditary-angioedema treatment. Intellia has also started licensing its CRISPR technology to other biotechs, such as the French biotech SparingVision to treat certain eye diseases and the California cell-therapy company Kyverna Therapeutics to focus on autoimmune diseases. The early data showed few and mild side effects and a dramatic reduction in levels of the disease-causing protein. Latest news: Intellia published preliminary results in June from the first six patients treated with its in-the-body CRISPR program. Those programs target sickle-cell disease (in partnership with Novartis) and a type of blood cancer called acute myeloid leukemia. The other two programs require extracting patients' cells, editing them in a lab with CRISPR, and reinfusing the modified cells. Two of those are CRISPR therapies delivered inside the body that treat a genetic nerve disorder called transthyretin amyloidosis and a genetic swelling disorder called hereditary angioedema. ![]() What Intellia's working on: The company has started early human testing for four programs. The firms on the list that have public or private valuations are worth more than $25 billion combined. It's a fast-growing area - nearly all these companies didn't exist just a decade ago. With a busy 2022 ahead for the gene-editing industry, Insider identified the top 18 companies to know that are leading the space. Altogether, the space is moving rapidly, with human gene-editing trials underway for a variety of diseases, including certain types of cancer, sickle-cell disease, and HIV. Other startups are building more advanced tools to edit genes, such as base editing and prime editing, which can edit genetic code more precisely and without breaking strands of DNA. Some of the gene-editing biotechs are working on the original CRISPR technology - CRISPR-Cas9 - that won a Nobel Prize in 2020. "Some may be expansions with the current companies. ![]() "I certainly believe there are more deals to come," Dolsten told Insider in a January 10 interview. Mikael Dolsten, Pfizer's top scientist, echoed that sentiment, suggesting the pharma giant wouldn't stop with the Beam deal. Account icon An icon in the shape of a person's head and shoulders.
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