Three gene therapy pioneers just won the Breakthrough Prize. This is their story

Ryan Cross / endpoints - A trio of scientists behind the first gene therapy approved in the United States have been awarded a Breakthrough Prize in Life Sciences. It’s arguably one of the most prominent recognitions yet for the field ...

AI Summary: Three gene‑therapy researchers received the Breakthrough Prize in recognition of foundational work that enabled the first CRISPR-based sickle cell therapy. The award highlights decades of basic and translational research that turned genome editing from lab curiosity into a tangible clinical remedy, with winners sharing a multimillion‑dollar prize and public acclaim.

First-Line Zongertinib in Advanced HER2-Mutant NSCLC: A New Targeted Standard Begins to Take Shape

oncodaily - For years, HER2-mutant non-small-cell lung cancer sat in an uncomfortable place in thoracic oncology. It was clearly an oncogene-driven disease, but unlike EGFR-mutant or ALK-rearranged NSCLC, it did not have […]

AI Summary: Clinical trial data show zongertinib delivers strong antitumor activity as a first‑line treatment for patients with advanced HER2‑mutant non‑small cell lung cancer, positioning the drug as a potential new targeted standard. Robust response rates in previously untreated patients are prompting clinicians to rethink initial treatment choices while attention turns to long‑term safety and access.

Growing liver tissue directly in the body could ease donor organ shortage

medicalxpress - In patients developing end-stage liver disease, the damage has become too severe for the liver's normally extraordinary regenerative capacity to repair or compensate for it. Once this "point of no return" has been reached, the only option is an organ tran…

AI Summary: Scientists report a technique to grow liver tissue directly inside the body as a potential solution to donor organ shortages, demonstrating functional hepatic tissue formation in preclinical or early clinical models. The promising results have been followed by an editorial expression of concern over methodology and data, prompting calls for independent validation before wider clinical use.

First-in-human trial primes immune system to accept donor livers

medicalxpress - UPMC and University of Pittsburgh clinician-scientists have weaned and kept multiple liver transplantation patients off of all immunosuppressant drugs for more than three years through a first-in-human clinical trial of a unique "immune priming" therapy.

AI Summary: A first‑in‑human study reports a novel immune‑priming approach that enabled three liver transplant recipients to stop standard lifelong immunosuppression. The intervention appears to induce tolerance to donor tissue, reducing dependence on anti‑rejection drugs and their toxic side effects, offering a potential shift in transplant management if larger trials confirm these early results.

ESMO 2026 Guideline: Redefining the Management of Metastatic Colorectal Cancer

oncodaily - Metastatic colorectal cancer (mCRC) represents a complex and biologically diverse disease in which treatment decisions increasingly depend on molecular characteristics, disease burden, and patient-specific factors. While therapeutic options have expanded …

AI Summary: The European Society for Medical Oncology released updated clinical practice guidelines for metastatic colorectal cancer, redefining diagnostic pathways, systemic therapy sequencing, and follow‑up standards. The guidance incorporates recent targeted and immunotherapy evidence to recommend practical treatment algorithms, prompting oncology centers to update protocols and payers to reassess coverage as clinicians translate recommendations into routine care.

FDA approves Travere's Filspari as first drug for the kidney disease called FSGS

Nicole DeFeudis / endpoints - The FDA expanded the label for Filspari on Monday to add another kidney condition. The drug is now the first therapy approved in the US for focal segmental glomerulosclerosis (FSGS). The pill may be taken ...

AI Summary: The FDA approved Filspari for focal segmental glomerulosclerosis (FSGS), delivering the first specifically authorized treatment for this rare kidney disease. The approval provides a targeted therapeutic option for patients and marks a commercial milestone for Travere, raising hopes for better outcomes while spotlighting questions about access, pricing, and long‑term real‑world effectiveness.

Norway's 'Oslo patient' reaches HIV remission after rare stem cell transplant donated by brother

medicalxpress - A Norwegian man has been effectively cured of HIV after receiving a stem cell transplant from his brother, doctors announced on Monday.

AI Summary: A remarkable clinical case: a patient in Norway known as the "Oslo patient" has entered sustained HIV remission following a rare stem‑cell transplant from a donor with genetic resistance to the virus. The outcome offers cautious optimism for curative strategies, though experts stress this remains an exceptional, high‑risk intervention rather than a broadly applicable cure.