STAT+: Astellas retries XLMTM gene therapy after deaths

STAT PlusNewsletterThe Readout Astellas retries XLMTM gene therapy after deaths Veradermics’ hair loss pill late-stage trial success, FDA fast-tracks reviews for psychedelics, and more biotech news Manage alerts for this article Email this article Share this article By Meghana KeshavanApril 27, 2026 Biotech Correspondent Meghana Keshavan[email protected]Meghana Keshavan covers biotech and contributes to The Readout newsletter. Want to stay on top of the science and politics driving biotech today? Sign up to get our biotech newsletter in your inbox. New hair loss data for a sort of amped-up minoxidil is looking very plush. Also Astellas is reentering the clinic with a next-generation gene therapy for XLMTM and Intellia’s CRISPR therapy for hereditary angioedema impresses.Advertisement The need-to-know this morning Eli Lilly said it is buying Ajax Therapeutics, a privately held developer of blood cancer drugs, for as much as $2.3 billion. Ajax’s lead medicine is a next-generation JAK2 inhibitor currently being evaluated in a Phase 1 study in patients with myelofibrosis. Ligand Pharmaceuticals said it is buying Xoma, an aggregator of drug royalties, for $739 million. Compass Therapeutics said its drug for advanced biliary cancer delayed tumor progression but failed to prolong survival compared to chemotherapy in a clinical trial. BridgeBio shares were higher this morning on reports from the weekend that Pfizer had settled two of three patent cases regarding Vyndamax, its medicine for the heart condition known as ATTR-CM. While terms of the settlement have not been disclosed, any extension of patent protection for Vyndamax is also positive for BridgeBio, which sells a competing ATTR-CM drug called Attruby. CRISPR therapy slashes rare swelling attacks Intellia Therapeutics said this morning that its one-time CRISPR treatment lonvo-z cut attack rates by 87% in a Phase 3 trial of hereditary angioedema, STAT’s Jason Mast writes. With more than 60% of patients becoming attack-free and no serious safety signals reported, the therapy is being positioned as a functional cure — or at least a major upgrade over existing options. STAT+ Exclusive Story Already have an account? Log in This article is exclusive to STAT+ subscribers Unlock this article — plus in-depth analysis, newsletters, premium events, and news alerts. Already have an account? Log in Monthly $39 Totals $468 per year $39/month Get Started Totals $468 per year Starter $30 for 3 months, then $399/year $30 for 3 months Get Started Then $399/year Annual $399 Save 15% $399/year Get Started Save 15% 11+ Users Custom Savings start at 25%! Request A Quote Request A Quote Savings start at 25%! 2-10 Users $300 Annually per user $300/year Get Started $300 Annually per user View All Plans To read the rest of this story subscribe to STAT+. Subscribe Log In biotechnology, drug development, Drug prices, research Submit a correction requestReprints Meghana Keshavan Biotech Correspondent Meghana Keshavan covers biotech and contributes to The Readout newsletter. Newsletter Your morning rundown of the science, politics, and money driving biotech today Recommended Biotech April 27, 2026 STAT Plus: Intellia says CRISPR-based treatment for rare disease reduced swelling attacks in pivotal trial Biotech April 27, 2026 STAT Plus: Oruka’s long-acting psoriasis therapy posts strong results in mid-stage study Advertisement Biotech April 27, 2026 STAT Plus: Veradermics’ hair loss drug succeeds in late-stage trial Biotech April 24, 2026 STAT Plus: FDA to speed up review of three psychedelics as mental health treatments The Readout April 24, 2026 STAT Plus: Sanofi MS drug gets positive review in Europe following FDA Subscriber Picks