Prosensa, GSK는 엑손스키핑 프로그램 확장!!

다른 삭제 유전자의 스키핑프로그램이 추가되었습니다.

• Prosensa는 dystrophin 유전자의 특정 exons (섹션)을 건너뛰는 기반으로 Duchenne 근육질 영양 장애 (DMD)에 대한 잠재적 인 치료제의 자사 포트폴리오를 확장했다.  엑손 - 스키핑 프로그램의 일부는 GlaxoSmithKline (GSK)와 협력하여 실시하게 된다. exons에게 51 (drisapersen)과 44 (PRO044)을 대상

• 화합물은 DMD에 대한 임상 실험에 있습니다. exons 52 55 대상

• 화합물은 최대한 빨리 임상 시험으로 이동합니다. exons 45 53 타겟

• 화합물은 유럽위원회 (European Commission)에서 고아 약물 지정을받은하고 다음 6 개월 이내에 임상 개발을 입력 할 것으로 예상됩니다.

계속 확장되고 있습니다.

원문나갑니다.

Compounds targeting exon 52 and exon 55 of the dystrophin gene as a way to treat some forms of Duchenne muscular dystrophy (DMD) will be moved into clinical trials as soon as possible, says Dutch biotechnology company Prosensa. The experimental compounds are known as PRO052 and PRO055.

Exons are sections of a gene. In DMD, the goal of the experimental treatment strategy known as exon skipping is to cause muscle fibers to leave out ("skip") flawed genetic instructions in the dystrophin gene and piece together instructions that can lead to production of a functional dystrophin protein. Dystrophin is the muscle protein that's missing in people with DMD.

The company also says it has received orphan drug designation from the European Commission to develop compounds targeting dystrophin exon 45 (PRO045) and exon 53 (PRO053). This type of designation gives companies financial incentives to spur development of treatments for rare disorders. These two compounds are expected to enter clinical development within the next six months.

Prosensa announced both developments in an Oct. 23, 2012, press release.

PRO051, PRO044 already in clinical trials

The company has developed two additional exon-skipping compounds that are already in clinical trials for DMD.

One, known as drisapersen (formerly known as PRO051/GSK2402968), targets dystrophin in exon 51 and is now in phase 3 clinical trials under the auspices of pharmaceutical company GlaxoSmithKline (GSK).

A phase 2 trial of drisapersen is open to recruitment at centers throughout the United States for boys with DMD who are at least 5 years old, able to walk, and have dystrophin mutations amenable to treatment by skipping exon 51. See Clinical Study to Assess Two Doses of GSK2402968 in Subjects with Duchenne Muscular Dystrophy (DMD114876); or enter NCT01462292 in the search box at ClinicalTrials.gov. You also can contact the U.S. GSK Clinical Trials Call Center at (877) 379-3718 or send an email to GSKClinicalSupportHD@gsk.com. Refer to study number NCT01462292 in your communication.

A phase 3 trial of drisapersen is ongoing at 45 centers outside the United States but is no longer recruiting participants. See Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects with Duchenne Muscular Dystrophy (DMD114044); or enter NCT01254019 in the search box at ClinicalTrials.gov.

Prosensa's PRO044, targeting exon 44 of the dystrophin gene, is being tested in a clinical trial in Europe that is no longer open to new participants. See Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy; or enter NCT01037309 in the search box at ClinicalTrials.gov.