Tag: gene therapy

November 7, 2023

AVB-101 will now benefit from the FDA’s fast track process, which is designed to improve the efficiency of product development and accelerate the review of treatments for serious conditions. FTD, a form of early-onset dementia, is the leading cause of […]

November 2, 2023

The potential therapy is the first to emerge from CRISPR’s and Vertex’s strategic partnership, which was expanded in 2021. Affecting more than 20 million people worldwide, SCD is a group of inherited red blood cell disorders which cause red blood […]

October 31, 2023

A decade ago, scientists outlined the gene editing potential of CRISPR, turning the vestiges of a bacterial immune system into one of the biotechnology industry’s most powerful tools. On Tuesday, a group of advisers to the Food and Drug Administration […]

October 31, 2023

Launched in 2012 under the US Food and Drug Administration’s (FDA) Safety and Innovation Act, Rare Pediatric Disease PRV’s specifically target and support the development of new drugs and biological products for certain, rare paediatric diseases. Upon approval, the rare […]

October 30, 2023

Patients with sickle cell disease may soon have two new treatments to try. On Tuesday, a U.S. Food and Drug Administration advisory committee will weigh the merits of a new gene therapy for the painful, inherited condition, which typically strikes […]

October 26, 2023

Despite optimism for a stronger investment climate this year, many of the hurdles that faced young biotechnology companies in late 2022 have endured through 2023. Initial public offerings for drugmakers have remained slow, with only 18 biotechs making their public […]

October 25, 2023

The decision, which specifically applies to adults with relapsed or refractory (R/R) MDS with an IDH1 mutation, makes Tibsovo the first targeted therapy approved for this indication. The US regulator has also approved the use of a companion diagnostic, the […]

October 24, 2023

When developing new medicines, drugmakers typically try to make treatments that are more effective, durable or safer than what’s already available. If they’re lucky, they can hit on something that meets at least one of those goals. Rampart Bioscience, a […]

October 12, 2023

The FDA has shown caution with companies developing RNA treatments for neuromuscular conditions. Dyne Therapeutics, Sarepta Therapeutics, Avidity Biosciences and Entrada Therapeutics have all faced delays in their work amid partial or full clinical holds. PepGen appears to have resolved […]

October 12, 2023

AstronauTx has announced the completion of a £48m Series A financing to create new treatments for Alzheimer’s disease (AD). As part of the investment led by the Novartis Venture Fund, the financing was supported by several global venture investors, including […]

October 5, 2023

Kyowa’s buyout of Orchard adds to a half dozen other gene therapy acquisitions over the past year. While the dealmaking is in some respects an encouraging sign, the sale prices have been modest and typically followed business setbacks for the […]

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 […]