| | | |   | Good morning, all. Damian here with promising news on a gene therapy that faces an uphill path to the market, plus some mRNA diplomacy and a pharmaceutical bakeoff. | | | Are gene therapies for ultra-rare diseases doomed? Researchers at the University of California, San Francisco have come up with a gene therapy for one of the rarest and hardest-to-treat forms of severe combined immunodeficiency, known as “bubble boy disease.” But despite promising data on the treatment’s benefits, the dismal history of commercializing such treatments for ultra-rare diseases has left scientists unsure how to get their medicine to patients who need it.
As STAT’s Brittany Trang reports, researchers have treated 10 patients whose severe combined immunodeficiency, or SCID, results from mutations to a gene called Artemis. All of the patients survived the therapy, and four no longer need immunoglobulin treatment. Some have even received childhood immunizations and created protective antibodies — a resounding success.
The question now is how to move forward with a treatment for a disease that affects only two to three newborns in the U.S. each year. UCSF researchers are hoping to find a biopharmaceutical company willing to shepherd its gene therapy to the market, but after witnessing the commercial failures of treatments for more common forms of SCID, they’re already looking into alternatives.
Read more. | mRNA makes Chinese landfall German authorities made their first shipment of mRNA Covid-19 vaccines to China, the government said yesterday, offering the latest hope that Western pharmaceutical companies might eventually find a market in the world’s second-largest economy.
The doses of BioNTech’s Pfizer-partnered vaccine will be available only to German expatriates, and their distribution is contingent on Germany clearing SinoVac’s Covid-19 vaccines for Chinese citizens living abroad. The development follows German Chancellor Olaf Scholz’s November trip to Beijing and the gradual dismantling of China’s strict “zero-Covid” policies.
Analysts and investors have become increasingly fixated on the possibility of selling mRNA vaccines in China, as BioNTech and its rival Moderna face steep revenue cliffs due to declining demand for their products. Both companies have repeatedly affirmed their eagerness to work with Chinese regulators to make their vaccines available there. Germany’s diplomatic success, while incremental, could be a prelude to Beijing softening its stance on Western vaccines. | Is boldness the solution to R&D challenges with bispecific antibodies? Despite their inherent challenges, bispecific antibodies (bsAbs) hold promise in improving the future of cancer care. T cell manipulation via bsAbs creates room for flexibility in immunotherapy research, with the ability to be studied individually as monotherapy or in combination with other oncologic treatments. Regeneron has spent over a decade researching bispecific antibodies, following the science to gain an understanding of the bsAbs landscape to utilize them in emerging research in oncology. Learn more. | Another biotech investor sells out HealthCor Management, a hedge fund focused on health care, is selling itself to private equity, the latest in a series of transactions that coincide with biotech’s lengthy downturn.
As STAT’s Allison DeAngelis reports, HealthCor will become part of Catalio Capital Management, launched in 2020. The deal, which consists mostly of stock, is expected to close early next year.
Catalio’s acquisition of HealthCor, which managed roughly $3.5 billion at its peak, caps a year full of investment M&A. In early 2022, European life sciences venture capital firm LSP was acquired by EQT AB for $450 million, and Abingworth, one of biotech’s oldest VCs, said in April that it would be acquired by Carlyle Group for an undisclosed sum. Apollo Global acquired a minority stake in Sofinnova Partners one month later.
Read more. | Pfizer is running a bakeoff in a $90 billion market Pfizer, a late-comer in the race to commercialize next-generation weight-loss drugs, is developing two drugs in search of one that might upend a market in the making.
The company has begun dosing in a mid-stage trial of the catchily named PF-07081532, partner Sosei said yesterday, enrolling about 800 patients with either Type 2 diabetes or obesity and testing its drug against both placebo and Novo Nordisk’s blockbuster semaglutide. The study, which is testing a range of doses, will help determine whether Pfizer moves forward with PF-07081532, a once-daily pill, or danuglipron, another mid-stage weight-loss drug that must be taken twice a day.
Both drugs spur weight loss by targeting GLP-1, a bodily protein that boosts insulin secretion. Pfizer estimates that the market for such medicines will exceed $90 billion by 2030, with prescriptions for obesity eventually outpacing those for diabetes. Novo and Eli Lilly currently dominate the market for GLP-1 drugs, but in-development medicines from Amgen, Pfizer, and others have the potential to offer more convenient dosing, greater efficacy, or fewer side effects. | More reads - Backed by a longtime mental health leader, a new startup aims to build support for serious mental illness, STAT
- Zynerba delays genetic disorder drug data on enrollment challenge, Reuters
- In a first, children with rare genetic diseases get mitochondrial transplants from their mothers, STAT
| Thanks for reading! Until tomorrow,  | | | |
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