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로슈, 다이노와 AAV 18억弗 파트너십..”CNS∙간 타깃” - 바이오스펙테이터

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로슈, 다이노와 AAV 18억弗 파트너십..”CNS∙간 타깃”

입력 2020-10-15 16:41 수정 2020-10-21 09:10

바이오스펙테이터 서윤석 기자

인공지능(AI) AAV 캡시드 발굴 플랫폼 ‘캡시드맵(CapsidMap)’ 이용, 면역원성, 표적화능력, 페이로드 제한등 기존 AAV 치료제 한계 극복

다이노(Dyno Therapeutics)가 로슈(Roche)와 함께 아데노바이러스(AAV) 유전자치료제 개발에 나선다. 다이노가 보유한 인공지능(AI) 기반의 AAV 캡시드 발굴 기술로 기존 AAV 유전자치료제의 한계를 극복하겠다는 컨셉이다.

다이노는 지난 14일(현지시간) 로슈와 중추신경계(CNS)와 간을 타깃하는 AAV 유전자 치료제 개발을 위한 파트너십을 체결했다고 밝혔다.

계약에 따라 다이노는 로슈로부터 비공개 선급금에 더해 개발단계에 따른 마일스톤으로 18억달러 이상을 받게 된다. 다이노는 페이로드 크기와 표적화 기능이 개선된 새로운 AAV 캡시드 설계를 담당한다. 로슈와 로슈의 자회사 스파크 테라퓨틱스(Spark Therapeutics)는 유전자치료제 후보물질에 대해 전임상부터 상업화 단계까지 개발을 담당한다.

아데노바이러스 벡터(AAV)는 유전자치료제 분야에서 치료제를 전달하는데 많이 사용된다. 체내에서 장기간에 걸쳐 안정적으로 치료유전자를 발현하면서도 면역반응이 거의 유도되지 않는 장점이 있다. 대표적인 AAV 유전자치료제는 노바티스의 척수성 근위축증(SMA) 치료제인 ‘졸겐스마(Zolgensma)’가 있다.

다이노는 인공지능(AI)을 이용한 머신러닝 기반으로 AAV벡터의 캡시드를 개선해 설계하는 캡시드맵(CapsidMap) 플랫폼 기술을 보유하고 있다. AAV 캡시드의 표적화 능력, 페이로드 크기제한, 면역원성, 제조특성 등을 개선해 기존 AAV 벡터의 한계를 극복할 수 있다는 설명이다. 캡시드는 바이러스의 껍데기 단백질로 세포를 투과해 세포내로 들어갈 수 있는 특징을 가지고 있다.

구체적으로 캡시드맵(CapsidMap)은 DNA 라이브러리에서 도출한 AAV 서열을 머신러닝과 고속분석(high-throughput) 방법으로 방대한 양의 실험 데이터와 분석, 평가해 새로운 AAV 캡시드를 빠르게 발굴하는 기술이다.

Eric Kelsic 다이노 CEO는 “로슈, 스파크와 협력해 중추신경계와 간을 타깃하는 유전자 치료제 개발로 영역을 확장하게 돼 기쁘다”고 말했다.

James Sabry 로슈 파트너링(Roche Pharma Partnering) 책임자는 “최적화된 AAV 벡터를 설계하는 다이노의 개발방식은 유전자 치료제를 보완해줄 것”이라며 “중추신경계와 간 치료제 개발을 통해 환자들에게 새롭고 혁신적인 치료제를 제공할 수 있기를 기대한다”고 말했다.

한편, 다이노는 노바티스, 사렙타와 각각 안구질환, 근육질환 관련 AAV 유전자치료제 개발을 위한 파트너십을 체결한바 있다.

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