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체내(in vivo) 유전자편집 CRISPR 치료제 첫 임상 개시 - 바이오스펙테이터

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체내(in vivo) 유전자편집 CRISPR 치료제 첫 임상 개시

입력 2019-07-26 15:45 수정 2019-08-02 07:29

바이오스펙테이터 봉나은 기자

이 기사는 '프리미엄 뉴스서비스 BioS+' 기사입니다.
엘러간-에디타스메디신, 레베르선천성흑암시(LCA10) 환자 대상 CRISPR 치료제 ‘AGN-151587’ 적용하는 Brilliance 임상1/2상 개시..환자 체내에서 직접 유전자 편집하도록 하는 in vivo CRISPR 치료제 첫 임상

체외(ex-vivo)가 아닌, 체내(in vivo)에서 유전자를 직접 편집(Genome editing)하는 CRISPR 치료제가 환자를 대상으로 한 첫 임상에 돌입했다. 엘러간(Allergan)과 에디타스 메디신(Editas Medicine)은 레베르 선천성 흑암시(LCA10) 환자를 대상으로 Brilliance 임상1/2상(NCT03872479)을 개시했다고 지난 25일 발표했다. 에디타스 메디신이 개발해 2017년 엘러간과 공동 개발하기로 계약한 CRISPR 기반 유전자 편집 치료제 중 하나인 ‘AGN-151587(EDIT-101)’에 대한 임상이다.

이번 임상은 CRISPR 기반 유전자 편집 치료제의 편집활동이 환자의 체내에서 일어나는 in vivo 방식의 첫 임상이라는 점에 의미가 있다.

중국이 2016년 처음으로 CRISPR/Cas9을 이용해 유전자 편집과정을 거친 세포를 환자에게 주입하는 임상을 시작하고, 이어 크리스퍼 테라퓨틱스(CRISPR Therapeutics)와 버텍스 파마슈티컬스(Vertex Pharmaceuticals)가 2018년 캐나다와 유럽에서 CRISPR/Cas9 유전자 편집 치료제의 임상을 시작했지만, 이들은 모두 ex-vivo 방식의 유전자 치료제를 사용했다. 체외에서 유전자 편집 기술로 환자 세포의 유전자를 교정해 다시 환자에게 주입했다는 말이다. 반면, 엘러간과 에디타스가 개발중인 AGN-151587은 환자의 체내에서 유전자 편집활동이 이루어진다는 점에서 의미가 다르다.

AGN-151587은 황색포도상구균 유래 Cas9인 ‘SaCas9’ 단백질을 암호화하는 DNA와, 2개의 ‘guide RNA’가 담긴 AAV5 벡터로 구성됐다. 이들이 환자 체내의 광수용체 세포(Photoreceptor cell)에서 발현돼, 유전자 변이를 제거하거나 전환시켜 정상적인 CEP290 단백질이 발현될 수 있도록 돕는다....

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