Tag: gene therapy

September 21, 2023

On Sept. 20, 2023, Alexion, AstraZeneca’s rare disease group, completed the purchase and license agreement for a portfolio of preclinical rare disease gene therapy programs and enabling technologies from Pfizer. The acquisition cost AstraZeneca up to $1 billion, with the […]

September 20, 2023

Taysha is now retrenching and focusing on another experimental drug, TSHA-102, for a rare neurological disorder called Rett Syndrome. The company now expects to reduce operating expenses enough to extend its cash runway into the fourth quarter of 2025. While […]

September 14, 2023

AlveoGene sprung out of the work of the U.K. Respiratory Gene Therapy Consortium, a group originally founded in 2001 to find a gene therapy for cystic fibrosis. In the process of developing a viral vector to deliver the cystic fibrosis […]

September 11, 2023

Novartis has stopped work on a gene therapy candidate it acquired a little under two years ago, according to an investor in the company the Swiss drugmaker bought. The decision to discontinue research follows a recommendation from a data monitoring […]

September 5, 2023

Success in the clinic would boost the RNA editing field as well as Wave. Broadly speaking, the technology is designed to edit specific sites in an RNA transcript without making permanent changes to a person’s genome. It’s an approach that’s […]

September 5, 2023

The funding will boost the University of Nottingham spinout’s development of bone regeneration treatment TherageniX, a company developing a dry powder gene therapy for bone graft augmentation, and the institution from which it emerged, the University of Nottingham, have been […]

August 31, 2023

Jeff Ajer, BioMarin’s executive vice president and chief commercial officer, said the announcement “represents an important milestone” for the haemophilia community, as well as for patients and physicians around the world seeking access to the therapy. Haemophilia treatment centres in […]

August 24, 2023

Royalty Pharma, a company specializing in acquiring pharmaceutical royalties, and Ferring Pharmaceuticals, a biopharmaceutical company with a new gene therapy expected to hit the market, announced that Royalty Pharma had acquired a synthetic royalty on US net sales of Ferring’s […]

August 22, 2023

Women may soon have a vaccine they can take during a pregnancy to help protect their newborn from respiratory syncytial virus (RSV), following U.S. Food and Drug Administration approval of the shot, called Abrysvo, on Monday The vaccine is designed […]

August 21, 2023

After the success of cancer immunotherapies like Keytruda and Opdivo, drugmakers went on a hunt to find new ways to defeat cancer by turning the immune system onto tumor cells. One such pathway was CD47, a cell protein that sends […]

August 16, 2023

The FDA’s decision to skip an advisory panel may indicate that agency reviewers are more unified in their views of the Bluebird therapy. Or it could simply indicate a higher comfort level with the type of treatment Bluebird is developing; […]

August 2, 2023

Former Biogen executive Michael Ehlers built Intergalactic with a vision to create a different type of gene therapy that wouldn’t need viruses as delivery systems. His team believed they could instead use electroporation, a method that uses electrical fields to […]

July 28, 2023

Once a bystander in the field of genetic medicine, AstraZeneca has started to make its ambitions clear over the last couple years. The company stuck to the sidelines, while drugmakers such as Novartis, Bristol Myers Squibb and Gilead made significant […]

July 26, 2023

Founded in October 2019, Kriya has lofty ambitions, describing its ultimate goal as “eliminating human suffering” from disease. CEO Shankar Ramaswamy says the company stands apart from rivals because its research platform can overcome challenges that have hampered the gene […]

July 24, 2023

In January 2022, the Milken Institute published a report, “Cell and Gene Therapies: Looking Ahead to 2022,”1 describing the growth of the CGT industry in 2021. In the U.S., 7 new therapies were approved for commercialization, a similar number were […]

July 20, 2023

FDA published a draft guidance document on July 13, 2023 outlining recommendations for manufacturing of products related to human cellular therapy or gene therapy (CGT). Recommendations included product compatibility and manufacturing changes regarding investigational and licensed CGT products. The draft […]

July 14, 2023

Caribou Biosciences – a company focused on genome-editing – has announced follow-up data from the dose escalation element of its ongoing ANTLER phase 1 trial. This new data set includes all 16 patients treated in dose escalation with the CB-010 […]

July 12, 2023

Avrobio was one of many gene therapy biotechs to launch in the latter half of the last decade, as scientific advances fueled a surge of interest and investment in medicines that use genes to treat disease. But the company’s prospects […]

July 5, 2023

Sarepta Therapeutics has sold the priority review voucher the Food and Drug Administration granted upon last month’s approval of Elevidys, its gene therapy for Duchenne muscular dystropy, the company announced Wednesday. The $102 million payment from an undisclosed buyer will […]

July 3, 2023

Artificial intelligence can predict on- and off-target activity of CRISPR tools that target RNA instead of DNA, according to new research published in Nature Biotechnology. The study by researchers at New York University, Columbia Engineering, and the New York Genome […]