Kyverna Therapeutics released one-year follow-up data showing patients with stiff person syndrome remained off other therapies after treatment with its CAR-T cell therapy miv-cel. The company plans to file for FDA priority review this year, which would make it the first cell therapy approved for an autoimmune disease.
The European Medicines Agency's Committee for Medicinal Products for Human Use has issued a positive opinion recommending Zeydovio (glepaglutide) for marketing authorization in treating short-bowel syndrome. The GLP-2 analog is developed by Zealand Pharma A/S.
The FDA granted full approval to Ultragenyx's gene therapy Fayuvi for Sanfilippo syndrome type A, marking the first-ever approved treatment for the ultra-rare, fatal neurodegenerative disease. The approval comes after a previous regulatory rejection and represents a significant milestone for patients with this devastating condition.
BrainChild Bio, spun out of Seattle Children's Hospital, secured $116 million in series A funding to advance its lead CAR-T therapy BCB-276 into pivotal trials for diffuse intrinsic pontine glioma, a rare and fatal brain tumor affecting approximately 300 U.S. children annually.
The U.S. Food and Drug Administration approved Mimrylo (rusfertide), developed by Takeda Pharmaceutical and Protagonist Therapeutics, as the first drug of its kind to treat polycythemia vera, a rare blood disorder that causes overproduction of red blood cells.
Silence Therapeutics announced that its Phase 2 SANRECO trial of divesiran met its primary endpoint in patients with polycythemia vera, a rare blood cancer, with data showing the treatment stabilized red blood cell levels and reduced phlebotomies to 0.2 per patient over 36 weeks. The company's stock rallied significantly on the news.
Intellia Therapeutics announced second quarter 2026 financial results alongside positive Phase 3 clinical data for lonvo-z in hereditary angioedema (HAE), with FDA biologics license application acceptance anticipated in late 2026 and U.S. commercial launch planned for first half of 2027.
Merck's anti-TL1A antibody from its 2023 Prometheus Biosciences acquisition delivered positive mid-stage results in hidradenitis suppurativa but failed to meet endpoints in a rare lung disease trial. The company disclosed the mixed Phase 2 results alongside its second-quarter earnings.
Britain's medicines regulator has approved Cytokinetics' drug for a rare heart condition, with the National Institute for Health and Care Excellence (NICE) recommending it for NHS reimbursement, making it accessible to approximately 6,600 patients in the UK.
Researchers have developed a new interventional treatment for hepatic myelopathy, a rare neurological complication of chronic liver disease that previously could only be cured through liver transplantation.
AstraZeneca announced Monday that its rare disease drug Ultomiris failed a late-stage trial for blood vessel complications following stem cell transplants, while a separate trial of its Claudin 18.2-targeting antibody-drug conjugate in stomach cancer succeeded. The Ultomiris setback adds to concerns about the drugmaker's pipeline following a series of setbacks this year.
Catherine L'Estrange from North Acton became the first patient in the UK and second in the world to receive groundbreaking gene therapy for Bardet-Biedl syndrome, a rare condition that progressively causes blindness in children. The treatment, performed at St Helier Hospital in March, involves injecting healthy copies of a gene directly into the eye.
Three major research institutions announced the creation of the Center for Therapeutic Genetics, a nonprofit collaboration aimed at developing standardized genetic medicines, including base and prime editing, for rare and ultra-rare diseases that currently lack treatments.
Two patients with SCN2A-related developmental epileptic encephalopathy experienced reductions in seizure frequency after receiving individualized allele-selective antisense oligonucleotides. The treatments were well tolerated, with no ASO-related serious adverse events reported in two parallel single-patient studies published in Nature Medicine.
A Northern California resident was hospitalized this spring after being confirmed as the fourth-ever person diagnosed with an extremely rare tick-borne disease caused by Rickettsia lanei bacteria, which produces symptoms similar to Rocky Mountain spotted fever. State health officials are warning residents to take precautions against ticks following the diagnosis.
Michael Larkin of Shirley, Long Island, survived New York's first confirmed case of Bourbon virus after being bitten by ticks while landscaping in May 2021. The rare tick-borne illness caused severe symptoms including high fevers, headaches, and rash, requiring a five-day hospitalization.
