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.

700-year-old mummy from Bolivia contains earliest confirmed evidence of strep throat bacteria in the Americas

livescience - A DNA analysis of pathogens from a pre-Hispanic mummy revealed that the bacterium that causes scarlet fever and strep throat was present in the Americas prior to European colonization.

AI Summary: Ancient DNA recovered from a 700‑year‑old Bolivian mummy has identified Streptococcus pyogenes—pathogen that can cause strep throat and scarlet fever—providing the earliest confirmed evidence of the bacteria in the Americas. Genomic analysis shows the bacterium was present well before European contact, reshaping timelines of infectious disease history and prompting fresh questions about pre‑Columbian pathogen ecology.

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.

Eli Lilly Buys Startup CrossBridge Bio to Bring a More Powerful Strike to Tumors

Frank Vinluan / medcitynews - Eli Lilly is paying up to $300 million for CrossBridge Bio, a startup developing antibody drug conjugates that deliver two drug payloads to cancers. Beyond potentially better efficacy, CrossBio’s dual approach could also fight drug resistance. The post El…

AI Summary: Eli Lilly agreed to acquire CrossBridge Bio for up to $300 million, adding small‑molecule oncology assets to its pipeline. The deal accelerates Lilly’s tumor‑targeting strategy, reflects Big Pharma’s preference for buying nimble biotech innovation, and highlights ongoing consolidation trends that determine where promising oncology programs ultimately end up.

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.

FDA to review whether to allow more access to certain peptides

medicalxpress - The U.S. Food and Drug Administration (FDA) will soon review whether certain peptides should be allowed in customized medications made by compounding pharmacies.

AI Summary: The FDA is moving toward expanding access to certain compounded peptides, opening a regulatory review that could loosen limits on how pharmacies and clinics obtain and prepare these popular therapies. The review balances potential patient demand and shortages against safety and quality-control concerns, putting compounding practices squarely under the spotlight.

Experimental drug cuts Parkinson's-linked protein up to 60% in early trial

medicalxpress - An experimental drug designed to silence a gene strongly linked to Parkinson's disease has shown encouraging effects in a first-in-human clinical trial, according to a study published in Nature Medicine. The drug, known as BIIB094, targets LRRK2, the most…

AI Summary: An experimental therapeutic cut levels of a Parkinson’s‑linked protein by up to 60% in an early human study, while complementary preclinical work identified a compound that clears toxic Parkinson’s proteins from brain tissue. The twin findings hint at disease‑modifying potential, though meaningful clinical benefit and long‑term safety remain to be demonstrated.

Insmed shelves Brinsupri in skin disease after mid-stage flop

Elizabeth Cairns / endpoints - Insmed has given up on Brinsupri in the painful skin disorder hidradenitis suppurativa (HS) after a mid-stage trial failure. The company had ditched the pill in sinus inflammation at the end of last year. The ...

AI Summary: Insmed announced it will halt development of Brinsupri for a dermatologic indication following disappointing mid‑stage trial results. Company commentary stressed the skin setback does not negate Brinsupri’s potential in pulmonary indications, but investors and clinicians will understandably ask for clearer signs of life before buying the comeback story.

Gilead Activates Its Pipeline: Option Exercises at Cartography Bio and Kymera Signal Accelerating Oncology Execution

oncodaily - In a pair of announcements that underscore Gilead Sciences’ intensifying commitment to oncology, two of its collaboration partners, Cartography Biosciences and Kymera Therapeutics, revealed today that Gilead has exercised exclusive […]

AI Summary: Gilead moved to accelerate its oncology strategy by exercising option agreements with smaller biotech partners, including Kymera and Cartography Bio. The moves signal targeted investment in protein degraders and other novel modalities, shoring up the company’s mid‑stage pipeline and hinting that big pharma still prefers buying options to inventing overnight.

Oricell Lands $110M to Take Cell Therapy to New Territory in Cancer

Frank Vinluan / medcitynews - There are no FDA-approved therapies for GPC3, a protein highly expressed by liver cancers. Oricell Therapeutics claims its cell therapy could be best in this class, but it faces competition from companies such as AstraZeneca and Eureka Therapeutics. The p…

AI Summary: Oricell, a China‑based CAR‑T developer, secured fresh financing to accelerate its cell‑therapy programs and support plans to go public. The fundraising will bankroll clinical expansion, manufacturing scale‑up, and regulatory preparations as the company pushes toward broader development and an IPO ambition, positioning it to compete in the crowded CAR‑T market.

FDA Approves Relacorilant with nab-Paclitaxel for Platinum-Resistant Epithelial Ovarian, Fallopian Tube, Or Primary Peritoneal Cancer

esmo - Evidence for efficacy is based on the findings from the ROSELLA study

AI Summary: The FDA granted marketing authorization for relacorilant in combination with nab‑paclitaxel to treat platinum‑resistant epithelial ovarian, fallopian tube and primary peritoneal cancers. Regulators cited clinical benefit in a difficult‑to‑treat population, providing an additional therapeutic option for patients with limited choices and marking an important regulatory milestone.

James P. Allison, PhD, FAACR, Honored With the 2026 AACR Award for Lifetime Achievement in Cancer Research

oncodaily - James P. Allison has been honored with the 2026 AACR Award for Lifetime Achievement in Cancer Research, a distinction recognizing his extraordinary scientific achievements and enduring impact on modern oncology. […]

AI Summary: James P. Allison received the 2026 AACR Award for Lifetime Achievement in Cancer Research for pioneering work in cancer immunotherapy. The award recognizes decades of transformative science that helped establish immune‑checkpoint blockade as a foundational cancer treatment, altering therapeutic strategies across multiple tumor types.

Antoni Ribas, MD, PhD, FAACR, Honored With the 2026 AACR-Margaret Foti Award

oncodaily - The American Association for Cancer Research (AACR) has named Antoni Ribas as the recipient of the 2026 AACR-Margaret Foti Award for Leadership and Extraordinary Achievements in Cancer Research, recognizing a […]

AI Summary: Antoni Ribas was honored with the AACR‑Margaret Foti Award in recognition of leadership and major contributions to cancer research. The award spotlights Ribas’ influence on immunotherapy development and cancer science translation, celebrating a career that has shaped both scientific agendas and clinical practice.