| | | |   | Hello, everyone. Damian here with an update on the titans of mRNA, what an AI coup means for medicine, and a long-awaited milestone for gene therapy. | | | BioNTech’s ‘historic chance’ to change biotech For Ugur Sahin and Ozlem Tureci, the husband-and-wife founders of BioNTech, the spoils of their Covid-19 collaboration with Pfizer present an opportunity not just to grow the company but to redefine just what a drug company can actually do.
Speaking to STAT’s Matthew Herper, Sahin and Tureci outlined their sweeping ambitions for BioNTech, which include mRNA-based treatments for cancer, infectious disease, and immunological disorders, focusing on both wealthy nations and low-income countries out of what Tureci called “a moral obligation to bring science fast to those who need it.”
Thanks to its Covid vaccine windfall, BioNTech has a “historic chance” to become a different kind of biotech, Sahin said, “a new generation of pharmaceutical company with multiple products in the market, with products not only in infectious disease and cancer but also in other disease areas. That is our target for 2030.”
Read more. | Merck bets $250 million on Moderna’s next act Elsewhere in mRNA, for the past two years, as Moderna has reaped billions in profit from its Covid-19 vaccine, Wall Street has grown increasingly antsy about just how the company might replace those revenues as demand dissipates. Yesterday, Merck offered a nine-figure endorsement of Moderna’s next act, a long-held plan to use its banner technology as a cancer treatment.
As STAT’s Adam Feuerstein reports, Merck is spending $250 million for the rights to mRNA-4157, a personalized cancer vaccine to be used in combination with its blockbuster medicine Keytruda. The two companies are running a clinical trial designed to determine whether the combination can delay the recurrence of skin cancer to a greater extent than Keytruda alone, and while results won’t be made public until later this year, the timing of the deal suggests Merck has seen enough to buy in.
The news sent Moderna’s share price up as much 12% yesterday, a move that likely had less to do with the dollar amount than the implications of a company like Merck seeing promise in something other than its Covid-19 vaccine. Moderna’s current $50 billion valuation is roughly half of what it was last fall, as disappointing demand for booster shots and a broader biotech downturn have dampened investor interest in the company’s future.
Read more. | How can sensitive molecular tools detect solid tumors sooner and inform precision treatments? Circulating tumor DNA (ctDNA) can yield an incredible wealth of information about cancer progression, response to treatment, and likelihood of recurrence, but key biomarkers are often lowly abundant. Find out how ultra-sensitive molecular methods are already driving advancements in therapy monitoring and minimal residual disease (MRD) testing for solid tumors, and discover use cases for technology platforms like next generation sequencing. | BioMarin’s gene therapy finally gets an FDA date After multiple regulatory delays and countless scientific debates, the first gene therapy for hemophilia could arrive in the U.S. this spring.
BioMarin Pharmaceutical said yesterday that the FDA had accepted its application for Roctavian, a one-time treatment for hemophilia A, and promised to make a decision on the medicine by March 23. The FDA rejected Roctavian in 2020, asking BioMarin to amass more data on its safety and long-term durability. European regulators approved it in August.
Approval for Roctavian would bring a long-awaited treatment option for patients with hemophilia A who need regular administrations of prophylactic treatment to ward off dangerous bleeding episodes. It would also mark a turning point for BioMarin, which expects to finally turn a consistent profit after 25 years in the red. | DeepMind’s AI wowed scientists. Can it actually lead to new drugs? It has been two years since DeepMind, a machine-learning company owned by Alphabet, dominated an annual competition in which computational biologists try to predict what proteins will look like in 3D given their amino acid sequence. The company has since modeled nearly every protein known to humankind, but the technology’s foundational promise to speed up the discovery of new medicines remains hotly debated among scientists.
As STAT’s Brittany Trang reports, experts liken DeepMind’s massive achievement to the Human Genome Project, as both a compliment and a criticism. Sequencing the human genome was a thunderclap in research, setting in motion countless new experiments. But the notion that it would lead to the rapid development of drugs for genetic disease was quickly dispelled by the complexity of biology.
Through that lens, experts said, DeepMind’s technology is less an answer in itself than a means of discovering new questions to ask. Janet Thornton, a professor at the European Bioinformatics Institute of the European Molecular Biology Laboratory, likened it to the James Webb Space Telescope: “There you can see the universe in a way that you didn’t before,” she said, “and I think we can see the protein universe in a more rounded way than we did before.”
Read more. | More reads - Differences in countries’ regulations keep some cell and gene therapies from reaching patients, STAT
- Novavax says COVID booster dose shows benefit against Omicron variants, Reuters
- Balwani denied request to join bid by Holmes for a new Theranos trial, Bloomberg
- Human neurons implanted in a rodent’s brain lead a rat to water — and make it drink, STAT
| Thanks for reading! Until tomorrow,  | | | |
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