Oxford Nanopore Technologies and Al Jalila Children's Specialty Hospital (AJCH) have launched a pilot program to diagnose undiagnosed rare disease patients. Through this collaboration, investigators will verify the effectiveness of long-read sequencing technology.
Oxford Nanopore Technologies와 Al Jalila Children’s Specialty Hospital(AJCH)이 미진단 희소 질환 환자 진단을 위한 파일럿 프로그램을 시작했습니다. 이번 협력을 통해 롱 리드 시퀀싱 기술의 효과를 검증할 예정입니다.
3billion signs MoU with Kolon Industries to jointly develop gene therapy for rare diseases. 3billion and Kolon Industries plan on conducting preclinical verification of drug candidates discovered through 3billion’s AI-driven drug development platform.
지난 9월 1일, 쓰리빌리언과 코오롱 인더스트리 미래기술원은 유전자치료제 개발을 위한 업무협약을 체결했습니다. 쓰리빌리언은 AI 기반 신약개발 플랫폼을 통해 발굴한 치료 후보 물질의 전임상 단계 검증을 코오롱인더스트리와 함께 진행할 계획입니다.
Novo Nordisk will pay $1.1 billion to acquire Forma Therapeutics. Novo Nordisk has recently been strengthening the treatment of rare blood disorders, while Forma Therapeutics has a drug for sickle cell disease in clinical trials.
Treatment for acid sphingomyelinase deficiency (ASMD), an ultra-rare disorder, has been approved for marketing by the FDA on August 31, 2022. Sanofi's treatment Xenpozyme was approved in Europe last June. ASMD has been referred to as Niemann-Pick Types A, A/B, and B. And it is known that there are approximately 120 patients in the United States.