The approval is a landmark moment for Sanfilippo patients who face progressive neurological symptoms and premature death.

The U.S. Food and Drug Administration (FDA) has granted approval to Fayuvi (rebisufligene etisparvovec-hopf), making it the first-ever gene therapy for pediatric patients with Sanfilippo syndrome Type

YouTube video by Sam and Esther Dolgoff Institute

Reviving the life and thought of the anarchist and scientist, Marie Goldsmith.

Marvel’s latest web-crawling adventure features a surprising dive into cutting-edge medicine, providing an opportunity for effective science communication.

Photographie de Marie Goldsmith (1873-1933) conservée aux AEN. Elle accompagnait une de ses lettres du 19 octobre 1916 cotée 55.21 avec la mention manuscrite suivante : “À notre ami James Guillaume. S...

World-leading vaccine and public health experts say NIH director Jay Bhattacharya’s recent op-ed is “infuriating” and “anti-scientific.”

BioXconomy and BPI investigate the booming underground peptide market – and the dangers it brings with it.

The clinical trial for the rare disease STXBP1 was originally put on hold following the death of a child.

Antibody drug developers are seeing regulatory wins and clinical trial successes for several different forms of cancer.

The regulatory body changed its opinion on antibody conjugate therapy Tivdak, paving way for NHS use.

Scientists have successfully used a base editing mRNA-LNP approach to correct genetic variants responsible for phenylketonuria (PKU), a severe metabolic condition. This non-viral gene editing method d

University of Barcelona develops ARI-0001, a not-for-profit CAR-T therapy using EU hospital exemption rules to improve patient access to cancer treatment.

Lipid nanoparticles have transformed the way biologics are delivered, creating robust and highly specific treatment options across a range of conditions.

The pharma giant says it has seen positive results from its BEHOLD-1 trial treating ovarian and endometrial cancer.

The collaboration will focus on the co-development of an AI antibody discovery platform.

New clinical trial results show promise for a monoclonal antibody immunosuppressant.

Lipid nanoparticles have transformed the way biologics are delivered, creating robust and highly specific treatment options across a range of conditions.

World-leading vaccine and public health experts say NIH director Jay Bhattacharya’s recent op-ed is “infuriating” and “anti-scientific.”

Researchers at two Chinese universities have shown that combination antibody therapies could prevent respiratory infections.

The US is driving innovation in the peptide therapeutics space, especially around GLP-1s.

A study of over 600,000 veterans suggests GLP-1s reduce the chances of developing addiction while improving outcomes for those already addicted.

Located in the village of Bonthapally, the peptide CMC site is set to open summer 2026.

A self-amplifying RNA with a “smart drug activation system” provides heart tissue-specific regeneration.

The US agency’s U-turn would postpone treatment for participants in the placebo group.

Academics innovate gene therapy manufacturing to cut costs and improve access, addressing commercialization challenges and saving lives worldwide

The pharmaceutical giant announced its candidate, tilrekimig, significantly reduced eczema severity in clinical trials.

Researchers from Kyoto, Japan, are advancing a drug that modulates the gut-brain axis with pro-cognitive effects across a range of indications.

mRNA delivered in vivo by antibody-targeted LNPs disrupts the defenses and microenvironment of pancreatic tumors.

Peptide-directed mRNA-LNP could treat reproductive disorders by thickening the uterine lining.

World-leading vaccine and public health experts say NIH director Jay Bhattacharya’s recent op-ed is “infuriating” and “anti-scientific.”

SDU dean Ole Skøtt said the trial will be assessed by ethics experts from the World Health Organization.

SDU dean Ole Skøtt said the trial will be assessed by ethics experts from the World Health Organization.

SDU dean Ole Skøtt said the trial will be assessed by ethics experts from the World Health Organization.