• A new study reveals a promising gene-editing approach that could one day treat many rare genetic diseases with a single, standardized method by helping cells ignore faulty “stop” signals in DNA and produce healthy proteins.
• Novartis’s new malaria drug, GanLum, worked extremely well in a major study, even against tough, drug-resistant strains, putting it on track to become the first new type of malaria treatment in decades.
• UC San Diego Health has become the first U.S. health system to offer a new blood test for testicular cancer that uses a microRNA biomarker to detect the disease with about 90% accuracy and guide treatment and follow-up more precisely.

Recent News

• New Gene-Editing Strategy Could Help Development of Treatments for Rare Diseases
  The New York Times – November 19, 2025
  Gene-editing therapies offer great hope for treating rare diseases, but they face big hurdles: the tremendous time and resources involved in devising a treatment that might only apply to a small number of patients. A study published recently outlines a new approach that could make the process more efficient and less costly. Writing in the journal Nature, researchers presented a path toward a gene-editing strategy that could eventually be standardized for many different rare diseases, instead of personalized edits for each one. “We are purposefully forgoing what is the most obvious way to treat a patient — fix their individual mutation back to the normal sequence,” said the study’s senior author, David R. Liu, a biologist at the Broad Institute and Harvard, who has pioneered several gene-editing advances. Instead, he said, the idea is a “disease-agnostic” strategy: developing a technique that “could treat many more patients regardless of what mutation they have.”
• Precise Bio completes first human cornea transplant using 3D-printed, lab-grown tissue
  Fierce Biotech – November 19, 2025
  Precise Bio has reported the first successful human implantation of its 3D-printed cornea implant, constructed of functional human eye cells cultured in a laboratory. The company described the procedure as the world’s first—and a major milestone toward its goal of alleviating the long wait times for people seeking transplants and faced with a lack of available donor tissue. According to North Carolina-based Precise Bio, its robotic bio-fabrication approach could potentially turn a single donated cornea into hundreds of lab-grown grafts. It estimates that there is currently only one available cornea for every 70 patients who need one to see.  “This achievement marks a turning point for regenerative ophthalmology—a moment of real hope for millions living with corneal blindness,” Aryeh Batt, Precise Bio’s co-founder and CEO, said in a statement.
• Novartis, with study success, to seek approval of new kind of malaria drug
  BioPharma Dive – November 12, 2025
  There haven’t been any new treatments for malaria for so long, in part, because artemisinin-based drug therapies “have worked so well,” said Sujata Vaidyanathan, the head of Novartis’ Global Health Development unit, at a media briefing. Yet that effectiveness is beginning to slip. In recent years, the World Health Organization has warned of escalating resistance to these drug cocktails in many African countries. The agency initiated a strategy to respond to the emerging threat in 2022 in an effort to minimize the impact of future spread. By that time, Novartis had already long been working on a possible solution. Known as ganaplacide, it’s designed to disrupt a protein transport system these parasites need to survive inside red blood cells. That’s important, according to Vaidyanathan, because it enables the drug to work against a broad spectrum of pathogens and at different stages of their life cycles.
• New therapy delays progression of recurrent prostate cancer
  UCLA Health News – November 12, 2025
  Patients with recurring prostate cancer who were treated with a new PSMA-targeted radioligand therapy before stereotactic body radiotherapy (SBRT) went more than twice as long without their disease worsening compared with those who received SBRT alone, according to new clinical trial results from UCLA Health Jonsson Comprehensive Cancer Center researchers. Findings from the trial, published in the Journal of Clinical Oncology and presented earlier this year at the 2025 American Society for Radiation Oncology Annual Meeting in San Francisco, showed that men who received the radioligand drug went a median of 17.6 months without disease progression, compared with 7.4 months for those who received SBRT alone. This translated into a significant delay in the start of hormone therapy, which is often used to treat recurrent disease.
• Prasad, Makary unveil new FDA path for accelerating custom gene editing therapies
  Fierce Biotech – November 12, 2025
  In a highly anticipated article, FDA officials Vinay Prasad, M.D., and Martin Makary, M.D., outlined a novel regulatory pathway that could trigger a seismic shift in how bespoke gene editing therapies are developed and approved. The new program, called the “plausible mechanism pathway,” is designed to offer a new way to market for personalized therapies, Prasad and Makary said at the beginning of a New England Journal of Medicine article. The pathway is expected to significantly scale the recent success of a single-patient treatment for a patient known as “baby KJ” into a widely available regulatory approach and offer new hope for accessible treatments among the estimated 300 million patients with rare genetic disorders. The article is “light on specifics,” Kiran Musunuru, M.D., Ph.D., who was part of baby KJ’s care team and is a co-founder of gene editing biotech Verve Therapeutics, said in an emailed statement to Fierce Biotech.
• Novartis opens new manufacturing plant in Carlsbad
  Los Angeles Times – November 11, 2025
  Swiss drugmaker Novartis opened a new 10,000-square-foot manufacturing facility in Carlsbad to make cancer drugs, as part of its promised $23 billion investment push to build out its domestic U.S. facilities over the next five years. The plant will produce compounds needed for radioligand therapy (RLT), a form of precision medicine that enables the delivery of radiation directly on cancerous tumors while limiting damage to surrounding cells. “Radioligand therapy is a breakthrough we’ve unlocked at scale, made possible by reimagining how innovation reaches patients,” said Vas Narasimhan, chief executive of Novartis. “As the global leader in RLT for more than seven years, we’ve advanced this technology with a deep belief in its power to transform cancer care.” This Carlsbad manufacturing facility will be Novartis’ third radioligand therapy production site in the U.S., and will help meet future demand for doses for patients in Western states and Hawaii.
• Disease of 1,000 faces shows how science is tackling immunity’s dark side
  Associated Press – November 6, 2025
  Doctor after doctor misdiagnosed or shrugged off Ruth Wilson’s rashes, swelling, fevers and severe pain for six years. She saved her life by begging for one more test in an emergency room about to send her home, again, without answers. That last-ditch test found the Massachusetts woman’s kidneys were failing. The culprit? Her immune system had been attacking her own body all that time and nobody caught it. “I just wish there was a better way that patients could get that diagnosis without having to go through all of the pain and all of, like, the dismissiveness and the gaslighting,” she said. Wilson has lupus, nicknamed the disease of 1,000 faces for its variety of symptoms — and her journey offers a snapshot of the dark side of the immune system. Lupus is one of a rogues’ gallery of autoimmune diseases that affect as many as 50 million Americans and millions more worldwide – hard to treat, on the rise and one of medicine’s biggest mysteries.
• UCB Wins First FDA Approval for Ultra-Rare Mitochondrial Disease
  BioSpace – November 4, 2025
  The FDA signed off on the use of UCB’s combination of doxecitine and doxribtimine for the treatment of thymidine kinase 2 deficiency, marking the first-ever approved therapy for the ultra-rare condition. The drugs are delivered together in an oral solution and will be marketed as Kygevvi. Kygevvi is indicated for adults and children with thymidine kinase 2 deficiency (TK2d) in whom symptoms start at or before 12 years of age. UCB will make Kygevvi available on U.S. shelves in the first quarter of 2026. Affecting fewer than 2 patients per 1,000,000 people, TK2d is caused by a mutation in the TK2 gene, which under healthy conditions produces a protein that helps maintain the proper function of the mitochondria. In patients with TK2d, this organelle weakens over time and becomes unable to provide enough energy for cells, in turn resulting in muscle weakness across the body—a key symptom.
• First in the United States to Offer New Testicular Cancer Blood Test
  UCSD Health – November 3, 2025
  UC San Diego Health is the first health system in the country to offer a new blood test for testicular cancer, a major advancement in the treatment and surveillance of patients with the disease. Developed after a decade of research, the test addresses a critical diagnostic gap, providing physicians with a sensitive and specific biomarker that informs treatment plans to avoid both under- and over- treating the disease. “Being able to engage in translational research that radically impacts the way we care for patients with testicular cancer is incredibly rewarding,” said Aditya Bagrodia, MD, professor of urology at University of California San Diego School of Medicine and urologic oncologist at UC San Diego Health. “Developing this test in partnership with my colleagues represents years of dedicated work to bring a new form of personalized medicine to testicular cancer care.”