Recent News

• FDA proposes new system for approving customized drugs and therapies for rare diseases
  AP News – February 23, 2026
  Federal health officials laid out a proposal to spur development of customized treatments for patients with hard-to-treat diseases, including for rare genetic conditions that the pharmaceutical industry has long considered unprofitable. The preliminary Food and Drug Administration guidelines, if implemented, would create a new pathway for bespoke therapies that have only been tested in a handful of patients due to the challenges of conducting larger studies. The FDA announcement specifically mentions gene editing, although agency officials said the new approach could also be used by other drugs and therapies.
• Assemblymember Diane Dixon Introduces Resolution Recognizing Rare Disease Day in California
  Office of Assemblymember Diane Dixon – February 23, 2026
  Assemblymember Diane Dixon introduced Assembly Concurrent Resolution 132 (ACR 132), which recognizes February 28, 2026, as Rare Disease Day in California. The resolution reaffirms California’s commitment to raising awareness, supporting individuals and families affected by rare diseases, and promoting policies that enhance research, treatment, and access to care. While individual conditions are considered rare, rare diseases collectively impact over 30 million Americans – roughly 1 in 10 individuals. Creating hardships for children and families, rare diseases often take years to diagnose, and once diagnosed, patients are faced with the reality that over 90% of rare diseases lack FDA-approved treatments.
• FDA accepts filing for Moderna flu vaccine after swift rethink
  Fierce Biotech – February 18, 2026
  The FDA has accepted Moderna’s revised influenza vaccine filing days after controversially refusing to review the original submission, putting the biotech on track to win approval in time for the 2026-27 flu season. Moderna reported that the FDA issued a refusal-to-file (RTF) letter in response to a request for approval of its flu vaccine candidate, mRNA-1010. In the RTF, the FDA’s Vinay Prasad, M.D., said the agency wouldn’t review the submission because Moderna’s comparator didn’t reflect the best-available standard of care in the U.S. at the time of the study. Moderna met with the FDA to discuss the situation and, just over one week after disclosing the RTF letter, said the agency has accepted an amended application for review.
• Nanoparticle-based gene editing could expand treatment options for cystic fibrosis
  UCLA Health – February 17, 2026
  UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene into human airway cells, restoring key biological function in a laboratory model of cystic fibrosis and establishing a potential new path toward mutation-agnostic gene therapy for inherited lung diseases. The study, published in Advanced Functional Materials, shows that lipid nanoparticles — tiny fat-based particles widely used to deliver mRNA vaccines — can be engineered to carry the complex molecular cargo required for precise insertion of a large full-length gene into the genome without using viral vectors. “This work shows that we can package everything needed for precise gene insertion into a single, non-viral delivery system,” said Dr. Steven Jonas, senior author of the study and a member of the UCLA Broad Stem Cell Research Center.
• Scientists Test “Smart” Nanotech to Target Cancer Tumors with More Precision
  UC Davis Health — February 13, 2026
  UC Davis Comprehensive Cancer Center scientists are testing “smart” nanotechnology that could transform cancer treatment by delivering drugs directly into tumors while sparing healthy tissue. The research is being conducted at the cancer center’s new Experimental Therapeutics Laboratory. The lab team has designed transformable nanoparticles (ultrafine particles) that travel through the body as tiny particles and then reshape into nanofiber networks when reaching the cancer sites. These fibers cling to tumors but naturally fade away much more quickly in healthy organs, creating a built‑in targeting system.
• MSD receives FDA approval for Keytruda combo to treat ovarian cancer
  Pharmaceutical Technology – February 12, 2026
  MSD has received the US Food and Drug Administration (FDA) approval for Keytruda and Keytruda Qlex, along with paclitaxel, with or without bevacizumab, for ovarian cancer. The therapies are approved as second or third line treatment for adults with platinum-resistant ovarian, fallopian tube, or primary peritoneal carcinoma whose tumors express programmed death-ligand 1, as determined by an FDA-authorized test. The approvals stem from the Phase III KEYNOTE-B96 trial data, which was presented at the 2025 European Society for Medical Oncology (ESMO) Congress.
• FDA Approves Wearable Device to Treat Pancreatic Cancer
  Medscape – February 12, 2026
  The FDA has approved a first-of-its-kind wearable electrical device — Optune Pax (Novocure) — for patients with locally advanced pancreatic cancer, in combination with gemcitabine and nab-paclitaxel. Optune Pax delivers alternating electrical fields, known as tumor treating fields (TTFields), to the abdomen. TTFields work by disrupting the rapid cell division characteristic of cancer cells, causing cell death. Swiss-based maker Novocure said the approval is the first for locally advanced pancreatic cancer in nearly 30 years. To use the device, patients wear electrically insulated adhesive patches that are placed on the abdomen and connected to an electric field generator, which is carried in a specially designed bag.
• Novartis breaks ground on San Diego biomedical R&D site
  PharmaPhorum – February 9, 2026
  Novartis has started construction of a $1.1 billion biomedical research centre in San Diego, as part of a $23 billion capital investment programme in the US that includes seven new facilities. The 466,000 sq ft centre will serve as a hub to join Novartis’ current R&D sites in Cambridge, Massachusetts, and Basel, Switzerland, and will carry out “end-to-end discovery across key disease areas and technology platforms,” according to the pharma group. Along with the San Diego facility, Novartis has said it intends to build four new manufacturing plants for drug substances, finished dosage forms, and medical devices over the next five years – including a flagship hub in North Carolina – along with three radioligand therapy (RLT) facilities located in California, Florida, and Texas.
• How “Invisible” Vaccine Scaffolding Boosts HIV Immune Response
  Scripps Research — February 5, 2026
  One of the biggest hurdles in developing an HIV vaccine is coaxing the body to produce the right kind of immune cells and antibodies. In most vaccines, HIV proteins are attached to a larger protein scaffolding that mimics a virus. Then, a person’s immune system produces a range of antibodies that recognize different bits of those proteins. Often, however, some of those antibodies react not to HIV itself—but to the scaffold used to deliver the vaccine. Now, researchers at Scripps Research and the Massachusetts Institute of Technology (MIT) have developed a new kind of vaccine scaffolding made from DNA that the immune system ignores, eliminating these off-target antibodies.
• Clinical Trial Offers First-of-Its-Kind Heart Failure Treatment
  UC Davis Health — February 2, 2026
  Heart failure occurs when the heart muscle cannot pump enough blood to meet the body’s needs. About 6.7 million adults in the United States are affected, and about one‑third of adults are at risk of developing it. Treatment for heart failure relies on medications to improve heart function and relieve symptoms, but many patients continue to struggle with shortness of breath, difficulty doing routine activities and even frequent hospitalizations. Researchers in cardiovascular medicine at UC Davis Health want to change this. They are hosting a clinical trial, that offers patients a minimally invasive procedure that could reduce extra pressure in the heart and address the source of many of their symptoms.
• Novel Cancer Vaccine Receives FDA Orphan Drug Designation in Cutaneous Melanoma
  CancerNetwork — February 2, 2026
  The FDA has granted orphan drug designation (ODD) to IFx-2.0 for the treatment of patients with stage IIB to IV cutaneous melanoma, according to a press release from TuHURA Biosciences. The ODD is based on results from an early phase 1 trial, with previous results being reported in the American Association for Cancer Research journals. The study results found that IFx-2.0 was safe and led to no serious dose-limiting toxicities (DLTs). Additionally, if patients were refractory to a checkpoint inhibitor therapy, a clinical benefit was experienced during subsequent anti–PD-1 treatment. “Our current focus with IFx-2.0 is targeting completion of enrollment in our phase 3 study of IFx-2.0 in combination with [pembrolizumab (Keytruda)] for the first-line treatment of advanced or metastatic Merkel cell carcinoma.
• Novo’s CagriSema tops semaglutide in ph. 3 diabetes study
  Fierce Biotech – February 2, 2026
  Novo Nordisk’s GLP-1/amylin combo has topped blockbuster semaglutide in a phase 3 Type 2 diabetes trial, though CagriSema still didn’t hit the 25% weight loss mark that the Danish pharma had previously laid out for its next-gen metabolic asset. A once-weekly shot of CagriSema, which combines semaglutide with the experimental amylin receptor agonist cagrilintide, reduced patients’ blood sugar by 1.91% on average from a baseline of 8.2% and dropped their weight by 14.2% after 68 weeks, Novo announced on Feb. 2. Injectable semaglutide, better known as Ozempic and Wegovy, came up short with reductions of 1.76% and 10.2%, respectively. “By combining semaglutide and cagrilintide, we’re seeing superior outcomes in both blood glucose control and weight reduction beyond those achieved with each therapy individually,” Martin Holst Lange, M.D., Ph.D., Novo’s R&D chief, said in the release.