If given the green light by the FDA, a CAR T cell therapy from Kyverna Therapeutics would be the first approved treatment for stiff person syndrome. Scientists are now testing CAR T cell therapy in nearly a dozen autoimmune diseases linked to harmful B cells, including lupus, rheumatoid arthritis and a connective-tissue disease known as systemic sclerosis. Kyverna, the California-based company behind the SPS trial, is also running trials for neurological autoimmune conditions including generalized myasthenia gravis and progressive multiple sclerosis, or MS. CAR T cell therapies are transformative, Konig says. “They’re going to change the field — but there are risks.” · Another risk is that the once-vanquished disease will return. Doctors don’t know all the genetic and environmental conditions that trigger autoimmune disease. In 2017, the FDA approved the first CAR T cell therapies for two blood cancers, acute lymphoblastic leukemia and large B cell lymphoma. Soon after, scientists saw the first tantalizing hints that it might work for autoimmune diseases, too. In some cases, cancer patients with autoimmune disorders reported improvements in both conditions after treatment. At 40, Lynn Montgomery-Haga was diagnosed with stiff person syndrome, a rare autoimmune disease that can cause severe muscle spasms and rigidity. In 2024, she received an experimental CAR T cell therapy for her condition — then her mobility began to come back.Animations by Eric Pio; Edited by Damien S.

WILKES-BARRE — U.S. Rep. Rob Bresnahan, R-Dallas Township, announced Wednesday he has co-sponsored several pieces of legislation intended to speed up insurance approval processes and hold health insurers accountable when treatment doctors say patients need is delayed or denied. • H.R. 9734 — Protecting Patients from Automated Denials Act: which establishes requirements for the use of artificial intelligence in prior authorization denials by Medicare Advantage organizations. • H.R. 9192 — Prior Authorization Reform for Autoimmune and Blood Disorders Act: which addresses insurance coverage and prior authorization barriers involving drugs for autoimmune diseases and certain blood disorders. To combat the delays in necessary care, contributions to patients abandoning recommended treatment, and added time from doctors and their staff, Rep. Bresnahan cosponsored: • H.R. 3514 — Improving Seniors’ Timely Access to Care Act of 2025: which establishes requirements for prior authorization in Medicare Advantage, including electronic prior authorization, faster decision timelines, transparency requirements, and greater accountability for MA plans. • H.R. 2433 — Reducing Medically Unnecessary Delays in Care Act of 2025: which establishes stricter federal standards for prior authorization decisions made by Medicare contractors and private Medicare plans. This bill mandates that coverage denials must be based on clear, evidence-based clinical criteria developed with input from practicing physicians. “Insurance companies operate in a multi-billion-dollar market, but patients are not dollar signs,” Bresnahan said. “Doctors should not have to spend hours fighting with insurers over treatments they have already determined are medically necessary. We can protect taxpayer and premium dollars without letting insurance companies put profits ahead of care.

Fate Therapeutics has seen improvements in four patients with treatment-resistant systemic sclerosis who were dosed with | “We were not expecting to see clinical responses so early,” Vaneet Sandhu, M.D., rheumatologist and vice president of clinical development at Fate Therapeutics, told Fierce. Fate Therapeutics has seen improvements in four patients with treatment-resistant systemic sclerosis who were dosed with its off-the-shelf CAR-T cell therapy in as little as three months. Data presented (PDF) today at the International Society for Stem Cell Research (ISSCR) in Montreal come from an ongoing phase 1 basket trial encompassing other autoimmune diseases, such as lupus. So far, investigators have treated a total of 30 patients. “We were not expecting to see clinical responses so early,” Vaneet Sandhu, M.D., rheumatologist and vice president of clinical development at Fate Therapeutics, told Fierce. The trial expanded to include patients with systemic sclerosis after researchers saw promising data from the treatment of Lupus patients with FT819. The data from these three patients, presented during the American Society of Gene and Cell Therapy meeting in May, showed a clinical improvement in disease activity without worsening in other clinical measures and no CRS, ICANS or GvHD. “We were not expecting to see clinical responses so early,” Vaneet Sandhu, M.D., rheumatologist and VP of clinical development at Fate, told Fierce. The company plans to advance the candidate to a phase 1/2 study across a range of autoimmune diseases in combination with standard-of-care therapies and without immunodepleting chemotherapy, Fate said. Fate Therapeutics CAR-T Cell & Gene Therapy Clinical Data Research Biotech

Generalized Myasthenia Gravis Market ... Alexion AstraZeneca Rare Disease, Cabaletta Bio, UCB, Immunovant, Roivant Sciences, Cartesian Therapeutics, Merck KGaA, RemeGen, Regeneron Pharmaceuticals, and others. Generalized Myasthenia Gravis Clinical Trial Analysis... Generalized Myasthenia Gravis Market Insights, Epidemiology, and Market Forecast – 2036 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key gMG companies, including Novartis, Alexion AstraZeneca Rare Disease, Cabaletta Bio, UCB, Immunovant, Roivant Sciences, Cartesian Therapeutics, Merck KGaA, RemeGen, Regeneron Pharmaceuticals, and others. Generalized Myasthenia Gravis Clinical Trial Analysis New York, USA, June 02, 2026 (GLOBE NEWSWIRE) -- Myasthenia Gravis Clinical Trial Pipeline Expands as 25+ Companies Race to Redefine Myasthenia Gravis Treatment Landscape | DelveInsight · The myasthenia gravis clinical trial analysis report delivers important insights into ongoing research of 30+ pipeline myasthenia gravis drugs, clinical strategies, upcoming therapeutics, and commercial analysis. For further information on the myasthenia gravis cure research, reach out @ Medication for Myasthenia Gravis Treatment ... Myasthenia Gravis Market Insights, Epidemiology, and Market Forecast – 2036 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key myasthenia gravis companies, including Immunovant, Roivant Sciences, Merck KGaA, Novartis, Alexion AstraZeneca Rare Disease, Regeneron Pharmaceuticals, Cartesian Therapeutics, Kyverna Therapeutics, Dianthus Therapeutics, NMD Pharma, ImmunAbs, UCB, Johnson & Johnson, argenx, Amgen, and others. At the recently concluded 2026 American Academy of Neurology (AAN) Annual Meeting, held April 18–22 in Chicago, Illinois, neuromuscular expert Tuan Vu, MD, a professor in the Department of Neurology at the University of South Florida Morsani College of Medicine, presented new data from the pivotal trial. In March 2026, Rallybio Corporation announced that they have entered into a definitive agreement pursuant to which Rallybio would acquire Candid through a merger transaction. Candid has built a leading portfolio of TCE therapeutics for autoimmune disease spanning a wide spectrum of B-cell and plasma cell targets with ongoing clinical studies in over 10 indications.

Royalty Pharma, a PM Capital holding, is reshaping how breakthrough medicines are funded and developed. By Alex Warnaar, PM Capital Nevertheless, we see Royalty Pharma as a diversified and lower-risk way to gain exposure to the long-term upside potential of medicine development and the biotech industry. There are an estimated 6,000 to 10,000 rare diseases of which over 90 percent have no FDA-approved treatment.[16] Several hundred new rare diseases are discovered or described each year. In March 2026, Royalty Pharma announced a US$500 million deal with Johnson & Johnson to advance the development of JNJ-4804, an investigational medicine for autoimmune diseases.[12] The deal is significant because it shows giant pharmaceutical companies are starting to use royalty funding in their capital structure to fund medicine development. Such is Royalty Pharma’s reputation that securing funding from Royalty Pharma can be a valuable form of endorsement for emerging biotech companies, given its due diligence when funding new medicines and record in backing winners. Cystic fibrosis (CF) is a rare genetic disease that primarily affects the lungs and digestive system. Over 100,00 patients have been diagnosed globally, often within days of birth. Left untreated, patients with the disease can live relatively short lifespans.[8] In 2014, the Cystic Fibrosis Foundation described Royalty Pharma’s funding as a ‘transformational moment for … [17] Pharma Research, ‘New Report: 800 New Medicines in Development to Treat Rear Diseases, https://phrma.org/blog/new-report-nearly-800-new-medicines-in-development-to-treat-rare-diseases, Accessed 4 May 2026.