A paper published in The Lancet showed that four out of four young children with Leber congenital amaurosis 4 retinal dystrophy due to a genetic deficiency of Aryl-hydrocarbon-interacting protein-like 1 (AIPL1) benefited substantially from treatment with Meiragtx Holdings plc’s rAAV8.hRKp.AIPL1 gene therapy. The four children were enrolled in the first clinical trial cohort, but a further seven children treated also have shown benefit. The company intends to submit a marketing authorization application (MAA) in the U.K. under exceptional circumstances based on the results from the 11 treated children, ages 1 to 4, all of whom were legally blind at birth, but who gained visual acuity after four weeks following a single dose of the therapy.

Amid the chaos, focus on innovation, says Stifel’s Opler

As the flurry of executive orders continues to flow from the White House, leaving in its wake chaos and confusion across the overall health care sector, Tim Opler, managing director of the global health care group at investment firm Stifel, is taking the long view and urging the biopharma sector to continue focusing on innovation. “The biotech sector is a great success story,” he said during a Feb. 19 virtual salon hosted by Demy-Colton on navigating the industry’s financial markets in 2025, a year Opler predicts will be no exception to that success. “I expect to see more great companies progress this year, more drugs get to patients through approvals by various regulatory agencies and, most importantly, the pace of innovation in research continue to rise.” It’s a forecast that seems unusually upbeat as the industry prepares to grapple with proposed cuts to research funding and a government-wide reduction in force, including 5,200 Department of Health and Human Services (HHS) employees who have reportedly received notice, with an estimated 22% of those coming from the NIH, along with proposed tariffs on pharmaceuticals that could start at 25% and go higher.

Buyers found for Bluebird; $3 per share – and maybe more

Bluebird Bio Inc. has agreed to be acquired by funds managed by global investment firms Carlyle and SK Capital Partners LP, along with a team of biotech executives. Stockholders of Somerville, Mass.-based Bluebird will get $3 per share in cash and a contingent value right per share that entitles the holder to a payment of $6.84 in cash per value right if Bluebird’s product portfolio hits $600 million in net sales in any trailing 12-month period on or before Dec. 31, 2027. Shares of Bluebird (NASDAQ:BLUE) were trading at $4.36, down $2.68, or 38%.

Asia recap: Innocare, Akeso in phase III; Deepseek in Antengene

Several Asia biotechs this week — including Innocare Pharma Ltd., Akeso Pharmaceuticals Inc., Sanbio Co. Ltd. and Ascletis Pharma Inc. — unveiled the start of new late-stage clinical trials or interim findings from early-stage studies. Others announced efforts to ramp up AI use in novel drug R&D operations, including Antengene Corp. Ltd.’s plans to deploy Deepseek for pipeline development. HBM Holdings Ltd. also partnered with Insilico Medicine Inc. to develop a new AI-powered antibody application, and Insilico said it founded a new AI-founded geroprotector asset called ISN018-055.

Astrazeneca, Vertex, Axsome secure US FDA approvals in January

The U.S. FDA approved 12 drugs in January, falling below the 2024 monthly average of 19. Only three new molecular entities received approval, trailing the yearly average of just over four per month. Therapies approved in January included: Datroway (datopotamab deruxtecan) for unresectable or metastatic hormone receptor (HR)-positive, HER2-negative breast cancer from Astrazeneca plc and Daiichi Sankyo Co. Ltd.; Journavx (suzetrigine) for moderate to severe acute pain from Vertex Pharmaceuticals Inc.; and Symbravo (meloxicam and rizatriptan) for migraine with or without aura from Axsome Therapeutics Inc.

Clots and metastasis in cancer patients start in the lung

The lung and thrombosis may play a key role in cancer and metastasis progression, according to a collaborative study led by Cornell University scientists. In the nonmetastatic lung microenvironment of several cancer types, the development of a prothrombosis niche promotes metastasis formation through the release of small extracellular vesicles loaded with an integrin protein. Extracellular vesicles can go to certain organ sites and promote metastases,” lead author David Lyden told BioWorld. “A melanoma which might metastasize to the lung will send up off extracellular vesicles that then go to the lung, and not any other organs, promoting putting metastasis to that organ. And pancreatic cancer would send extra biliary vesicles predominantly to the liver,” he explained.

Also in the news

Alume, Apellis, Aptose, Assembly, Astrazeneca, Atai, Biorestorative, Boehringer Ingelheim, Boost, Catalent, Clearside, CSl, Fibrogen, Gate, Genmab, GSK, Harbour, Imunon, Insilico Medicine, Irlab, Max, Medivir, Metaba, Myris, Novadip, Photys, Polaritybio, Polymed, Sellas, Signet, Sobi, Terns, Thetis, Urogen, Yaqrit