Prime Medicine Receives Additional Funding from Cystic Fibrosis Foundation for Prime Editing Treatments

Prime Medicine has secured up to $24 million in additional funding from the Cystic Fibrosis Foundation to accelerate the development of Prime Editors for cystic fibrosis treatments. The funding builds on an initial agreement and will support the development of multiple hotspot Prime Editors that can correct a wide range of genetic mutations, potentially treating the majority of people with cystic fibrosis.

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) has received a significant boost in its cystic fibrosis treatment portfolio with the European Commission's approval of its new triple combination therapy, ALYFTREK (deutivacaftor/tezacaftor/vanzacaftor), on July 1, 2023 [1]. This approval comes after ALYFTREK demonstrated non-inferiority compared to the existing treatment, KAFTRIO, in terms of lung function measurement. The approval is expected to target over 31,000 people struggling with cystic fibrosis across the European Union, representing the broadest label for the medicine and strengthening the company's revenue base.

The approval of ALYFTREK is a significant milestone for Vertex, which has a history of innovation in cystic fibrosis treatments. The company's existing medicines are treating over 75,000 people with CF in more than 60 countries on six continents, representing approximately 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy [2].

In addition to the European Commission's approval, Vertex has also secured a broad reimbursement agreement with NHS England for ALYFTREK. This agreement comes after the National Institute for Health and Care Excellence (NICE) issued a positive final draft recommendation for the medicine. The agreement will ensure that eligible patients in England can access ALYFTREK, further expanding its reach [2].

Meanwhile, Prime Medicine has secured up to $24 million in additional funding from the Cystic Fibrosis Foundation to accelerate the development of Prime Editors for cystic fibrosis treatments. The funding builds on an initial agreement and will support the development of multiple hotspot Prime Editors that can correct a wide range of genetic mutations, potentially treating the majority of people with cystic fibrosis. This new funding will help Prime Medicine advance its innovative approach to gene editing and expand its impact on cystic fibrosis treatments [3].

References:
[1] https://finance.yahoo.com/news/eu-commission-approves-vertex-pharmaceutical-131042914.html
[2] https://www.lelezard.com/en/news-21887023.html
[3] Prime Medicine press release