Since the first gene therapy was approved 4 years ago, gene therapy has been highly anticipated. However, it has recently been stalled as many clinical trials have failed due to safety concerns. This year, attention is being focused on whether or not these problems can be overcome and a therapy can be successfully developed.
4년 전 최초의 유전자 치료제가 승인된 이후로, 유전자 치료제는 큰 기대를 모았지만 최근 안전성 문제로 많은 임상 시험들이 실패하면서 정체를 겪고 있습니다. 올해는 이런 문제를 극복하고 치료제가 성공적으로 개발될 수 있을지 주목받고 있습니다. Original Article: BioPharma Dive.5 questions facing gene therapy in 2022
A $2.1 million study will be conducted at the University of Newcastle in Australia to develop a treatment for cystic fibrosis. Cystic fibrosis is a rare respiratory disease that can be caused by more than 2000 genes.
According to a report by Global Genes and DealForma, rare disease drug development funding in 2021 was estimated at $22.9 billion, with an increase of 28% compared to 2020. The funding size was compared with the overall size of the various rare disease-related deals, such as venture capital, IPO, and M&A.
An exome sequencing study of 428 children with cardiomyopathy was reported by AJHG. In 32% of patients, the genetic cause of the disease was identified. Research is needed as more genes that are not yet known are expected to cause cardiomyopathy.
It is estimated that 30 million people are affected by rare diseases in the EU region. Since 2009, the EU has invested 1.8 billion euros over the last 14 years, and a new partnership in 2023 is expected to strengthen rare disease clinical treatment and research through data sharing and collaborative policies.