Rare disease diagnostics & research

  1. The Rady Children’s Institute for Genomic Medicine announced a collaboration with PlumCare RWE to expand its rWGS-based newborn screening program in Greece. PlumCare and the Greek National Public Health Organization plan to recruit at least 1,000 families for the pilot program

    Rady Children’s Institute for Genomic Medicne가 PlumCare RWE와 그리스에서 rWGS 기반 신생아 선별 검사 프로그램을 확대하기 위해 협력한다고 발표했습니다.  PlumCare는 Greek National Public Health Organization과 함께 최소 1,000명 이상의 가족들을 모집할 계획이라고 밝혔습니다.
    Original Article: 360Dx. Rady Children's, PlumCare RWE Partner to Expand WGS-Based Newborn Screening Program to Greece

  1. The FDA has approved medicine for Amyotrophic Lateral Sclerosis(ALS) developed by Amylyx Pharmaceuticals. This brings ALS patients to two drugs, along with riluzole, which was approved 25 years ago. Like the conventional drug, Amylyx's drug cannot cure ALS, but clinical trials have shown it to slow the progression of ALS symptoms and prolong survival.

    FDA가 Amylyx Pharmaceuticals가 개발한 ALS 치료제를 승인했습니다. ALS 환자들에게 적용할 수 있는 약물은, 25년 전 승인된 riluzole와 함께 2개가 되었습니다. 기존 약물과 마찬가지로 Amylyx의 약물 또한 ALS를 치료할 수는 없지만, 임상시험에서 ALS의 증상악화를 늦추고 생존 기간을 늘리는 효과를 보였습니다.
    Original Article: BioPharma Dive. ALS drug approved by FDA in closely watched decision, marking win for patients, developer

  2. AstraZeneca will acquire gene-editing company LogicBio for $68 million. AstraZeneca will buy all of LogicBio's outstanding shares, and the value of the shares is over 600% compared to the time before the acquisition announcement. LogicBio has the technology to edit genes with homologous recombination technology, not CRISPR-based technology.

    AstraZeneca가 유전자 편집 기업 LogicBio를 6천8백만 달러에 인수할 예정입니다. AstraZeneca가 LogicBio의 모든 발행주식을 인수하는 방식으로 진행되며, 주식 가치는 인수 발표전 시점과 비교해 600% 이상의 가치로 평가되었습니다. LogicBio는 CRISPR 기반 기술이 아닌, homologous recombination 기술로 유전자를 편집하는 기술을 보유하고 있습니다.
    Original Article: BioPharma Dive. AstraZeneca to acquire gene editing biotech LogicBio

Do you want to keep up with the latest post of this series?