Regeneron Pharmaceuticals is collaborating with CRISPR technologies startup Mammoth Biosciences to develop in vivo gene-editing therapies. The deal marks the third pharmaceutical industry partnership for Mammoth, which initially focused on developing CRISPR-based diagnostics.
Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is now approved in the United Kingdom for treating the blood disorders sickle cell disease and beta thalassemia. It’s the first regulatory approval in the world for a CRISPR-based therapy.
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Graphite Bio voluntarily paused a Phase 1/2 test of its gene-edited therapy for sickle cell disease after the first patient in the study developed a serious blood complication. The setback will also delay plans to reach the clinic with another genetic medicine in its pipeline.
Sanofi is collaborating with Scribe Therapeutics to use the startup’s CRISPR technology to create cell therapies based on natural killers, a type of immune cell that has cancer-killing capabilities. The pharma giant is paying Scribe $25 million up front to kick off the deal.
Intellia Therapeutics has encouraging early clinical data for a gene-editing therapy addressing the rare swelling disorder hereditary angioedema. This therapy, the biotech’s second CRISPR-editing based treatment that performs in vivo edits, offers potential for a one-time treatment of a disease that is currently addressed by chronic oral and injectable drugs.
Bristol Myers Squibb is partnering with Century Therapeutics, placing a $150 million bet that the biotech’s technology for engineering stem cells can produce new off-the-shelf cell therapies for cancer. If the four potential programs covered under the pact reach the market, milestone payments could bring Century more than $3 billion.
The future looks bright for the integrated specialty pharmacy model – for health systems, providers, and most importantly, for patients.
Biotech startup GenEdit is developing polymer nanoparticle technology to deliver genetic medicines, an approach intended to avoid the limitations of viral vectors. Already partnered with a clinical-stage company, it now has financial support from a big pharma giant that joined a syndicate of investors in a $26 million Series A round.
Mammoth Biosciences is applying CRISPR technology to both diagnostics and therapeutics. With the new financing, CEO Trevor Martin said that the company is looking ahead toward clinical trials and perhaps partnerships with larger companies.
Caribou Biosciences’ CRISPR approach to cell therapy sparked strong investor interest, enabling the clinical-stage biotech to upsize its IPO and raise $304 million in the biggest life sciences IPO this week. Absci, Sophia Genetics, and Cytek Biosciences also priced IPOs.
The Food and Drug Administration had previously granted an emergency use authorization for Abbott's ID NOW point-of-care test, a decision for which the agency came under fire amid reports of the test delivering inaccurate results.
As technology advances, AI-powered tools will increasingly reduce the administrative burdens on healthcare providers.
The companies plan to apply with the Food and Drug Administration for an emergency use authorization by the end of the year. Mammoth's technology is based on the research of CRISPR pioneer Jennifer Doudna.
The Cambridge, Massachusetts-based company, co-founded by CRISPR pioneer Feng Zhang, said Thursday that it had received an emergency use authorization for its SARS-CoV-2 test, which works by programming a CRISPR molecule to pick up the virus' genetic signature.
The companies plan to focus initially on development of two monoclonal antibody candidates, with the aim of initiating Phase II clinical development in the next three to five months.
The company was founded to develop base editing, a form of gene editing that alters genes at the single-letter level rather than using the "cutting" technique typical of CRISPR/Cas9. Beam had filed to go public in September, aiming at raising $100 million.
The latest round of funding, led by Chinese investors, brings the total amount of money raised by the company, co-founded by CRISPR pioneer Jennifer Doudna, to more than $70 million.