Gene therapy specialist Meiragtx Holdings plc is heading for its first marketing approval following the successful treatment of 11 children with Leber amaurosis, a severe form of congenital retinal dystrophy that rendered them blind at birth. The 11 children, aged between 1 and 4 years old, all gained visual acuity following a single delivery of a correct version of the AIPL1 (aryl-hydrocarbon interacting protein-like1) gene, which codes for a photoreceptor protein in the cones and rods.
The longstanding ambition of developing an inhaled gene therapy for cystic fibrosis has taken a step forward, with the start of a phase I/II trial of a product using a novel pseudotyped viral vector that it is hoped will circumvent problems encountered in previous studies with other vectors.
EG 427 SAS has closed a €27 million (US$28.3 million) series B round, which will fund it to completion of the first clinical trial of the lead gene therapy program, opening the way for its herpes simplex viral-vectored products to be developed in a range of chronic neuro-urology disorders.
The scientific establishment has launched a fight back against the Trump administration and the slew of executive orders that threaten to dismantle research funding, with a warning that the next month could be critical for the future of U.S. science.
The former head of the U.S. NIH has sounded a warning that the uncertainty caused by the Trump Administration’s funding cuts and layoffs is blocking “critical work” and “paralyzing” biomedical research. “Every time we launch a new program, every time we continue to commit resources to ongoing work, those are important decisions that we make every single day, and in times like this, that decision-making is paralyzed,” said Monica Bertagnolli, who stood down as director of the NIH on Jan. 17.
The former head of the U.S. NIH has sounded a warning that the uncertainty caused by the Trump Administration’s funding cuts and layoffs is blocking “critical work” and “paralyzing” biomedical research. “Every time we launch a new program, every time we continue to commit resources to ongoing work, those are important decisions that we make every single day, and in times like this, that decision-making is paralyzed,” said Monica Bertagnolli, who stood down as director of the NIH on Jan. 17.
The first clinical data from an FDA-approved trial of a CAR T-cell therapy in the treatment of multiple sclerosis (MS) show the cells cause complete B-cell depletion, leading on to a reset of the immune system that is accompanied by an improvement in symptoms.
The scientific establishment has launched a fight back against the Trump administration and the slew of executive orders that threaten to dismantle research funding, with a warning that the next month could be critical for the future of U.S. science.
The scientific establishment has launched a fight back against the Trump administration and the slew of executive orders that threaten to dismantle research funding, with a warning that the next month could be critical for the future of U.S. science.
New research has uncovered a complex interplay between extracellular matrix (ECM) structure and the transcriptional responses of cancer cells, showing how they alter their gene expression to ‘escape’ from ECM. The findings will make it easier to identify tumors that are likely to metastasize at an early stage.
