Urea cycle disorders(CAMP4 Therapeutics)

DBA is caused by genetic changes in ribosomal sub-units that stop the maturation of red blood cells. Credit: ANIRUDH on Unsplash. Fulcrum Therapeutics has signed a global exclusive licence agreement with CAMP4 Therapeutics to develop new therapies to treat Diamond-Blackfan anaemia (DBA), a rare blood disorder. Fulcrum will hold the licence to intellectual property arising from the DBA programme of CAMP4. Recommended Reports Reports Subacute Necrotizing Encephalomyelopathy (Leigh Disease) Drugs in Development by Stages, Target, ... GlobalData Reports Kennedy’s Disease (Spinal and Bulbar Muscular Atrophy) Drugs in Development by Stages, Target, Mo... GlobalData View all Companies Intelligence Fulcrum Therapeutics Inc Fulcrum Pty Ltd CAMP4 Therapeutics Corp View all The company will also carry out the discovery, development and marketing activities of new therapeutic agents against an undisclosed target for DBA. Fulcrum will make an undisclosed upfront payment to CAMP4, which is also eligible to receive $70m in milestone payments on meeting specific development, regulatory and commercial goals. Fulcrum will handle expenses linked to research, development, production and marketing, and related activities. The company will also make royalty payments to CAMP4 on future commercial product sales. Fulcrum Therapeutics chief scientific officer Jeff Jacobs stated: “We are deeply committed to bringing hope and new options to patients suffering from rare haematologic diseases and are excited to expand on the work of CAMP4’s pre-clinical DBA programme. “This agreement further strengthens our discovery pipeline and reinforces our strategy of addressing rare genetic conditions through small molecules.” DBA is caused by genetic changes in ribosomal sub-units that stop the maturation of red blood cells and result in anaemia. In June 2023, CAMP4 raised $100m in a Series B financing round to progress the development of regulatory ribonucleic acid-focused programmes.