“Scientists in China who are currently researching CRISPR for its potential to treat various genetic diseases by modifying cells other than embryos say that they fear He’s actions might have a chilling effect on their work too, even though it is not as ethically fraught,” the announcement stated. is a service of the American Association for the Advancement of Science. READ MORE: Gene Therapy, Regenerative Medicine Markets Booming Worldwide. You can read our privacy policy for details about how these cookies are used, and to grant or withdraw your consent for certain types of cookies. “The first patient dosed in the BRILLIANCE clinical trial marks a significant milestone toward delivering on the promise and potential of CRISPR medicines to durably treat devastating diseases such as LCA10. The two colleagues who assisted him faced a shorter jail sentence. The trial is sponsored by Allergan plc and Editas Medicine. 503-494-4158 Consent and dismiss this banner by clicking agree. Organization TypeSelect OneAccountable Care OrganizationAncillary Clinical Service ProviderBioMedical EngineeringBiotechnology CompanyClinical Research OrganizationFederal/State/Municipal Health AgencyHospital/Medical Center/Multi-Hospital System/IDNLife SciencesMedical Device ManufacturerOutpatient CenterPayer/Insurance Company/Managed/Care OrganizationPharmaceutical CompanyPhysician Practice/Physician GroupSkilled Nursing FacilityVendor, Sign up to receive our newsletter and access our resources. Additional academic institutions involved in the clinical trial currently include OHSU, Bascom Palmer Eye Institute in Miami, Florida, Massachusetts Eye and Ear in Boston, and WK Kellogg Eye Center at the University of Michigan in Ann Arbor, Michigan. by contributing institutions or for the use of any information through the EurekAlert system. The study uses the gene-editing technology CRISPR to repair mutations in the CEP290 gene that cause a rare form of inherited blindness called Leber congenital amaurosis type 10. “The growth of innovative research and product development in the field of gene therapy is exciting to us as physicians, scientists, and regulators,” said FDA Commissioner Stephen M. Hahn, MD, said in the announcement. Clinical researchers at Casey Eye Institute, Oregon Health & Science University (OHSU), have dosed the first patient with an experimental CRISPR/Cas9 therapy in the BRILLIANCE Phase 1/2 clinical trial for people with Leber congenital amaurosis 10 (LCA 10). EurekAlert! The trial is sponsored by Allergan plc. “Being able to edit genes inside the human body is incredibly profound,” said Mark Pennesi, MD, PhD, the Kenneth C. Swan, associate professor of ophthalmology in the OHDU School of Medicine and chief of the OSHU Casey Eye Institute’s Paul H. Casey Ophthalmic Genetics Division. Previously, gene-editing methods have edited genetic material after it was removed from the human body. Enter your email address to receive a link to reset your password, New Ruling to Expand Public Access to Clinical Trial Data, ©2012-2020 Xtelligent Healthcare Media, LLC. The study uses the gene-editing technology CRISPR to repair mutations in the CEP290 gene that cause a rare form of inherited blindness called Leber congenital amaurosis type 10. At the beginning of January, He Juankui was sentenced to three years in prison for “illegal medicine practice” in China after he announced that he created the world’s first gene-edited babies using CRISPR. The trial’s gene-editing is designed to be permanent but not passed onto the offspring of those who receive it through its gene editing approach. OHSU is dedicated to improving the health and quality of life for all Oregonians through excellence, innovation and leadership in health care, education and research. whitef@ohsu.edu The documents will further efforts to create a modern structure for gene therapy development.  @ohsunews, Copyright © 2020 by the American Association for the Advancement of Science (AAAS). Thanks for subscribing to our newsletter. Notice of Privacy Practices The BRILLIANCE Phase 1/2 clinical trial of AGN-151587 (EDIT-101) for the treatment of Leber congenital amaurosis 10 (LCA10) will assess the safety, tolerability, and efficacy of AGN-151587 in approximately 18 patients with this disorder. OHSU is an equal opportunity affirmative action institution. And Editas Medicine just recently announced that the first patient living with LCA10 was recently treated through the clinical trial. provides eligible reporters with free access to embargoed and breaking news releases. Juankui was in pursuit for “fame and profit” at the time that this situation occurred. © 2001-2019 OHSU. Credit: OHSU News Mark Pennesi, M.D., Ph.D., who leads OHSU's involvement in the trial, center right, looks on as staff at Oregon Health & Science University's Casey Eye Institute perform the first-ever in vivo CRISPR gene edit procedure for the BRILLIANCE clinical trial. ACTIV-2 was established as part of NIH’s Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV), a public-private partnership program instituted to speed development of the most promising treatments and vaccines. This outcome is promising, as patients currently living with this disease have had no treatment options prior, the company highlighted. Complete your profile below to access this resource. The Brilliance clinical trial is a Phase 1/2 study to evaluate AGN-151587 for the treatment of LCA10. BRILLIANCE trial. Franny White Mark Pennesi, M.D., Ph.D., who leads OHSU's involvement in the trial, center right, looks on as staff at Oregon Health & Science University's Casey Eye Institute perform the first-ever in vivo CRISPR gene edit procedure for the BRILLIANCE clinical trial. “As the regulators of these novel therapies, we know the framework we construct for product development and review will set the stage for continued advancement of this cutting-edge field and further enable innovators to safely develop effective therapies for many diseases with unmet medical needs,” explained Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research. The procedure uses gene-editing tool CRISPR to edit human genes, also known as in vivo gene editing. This may help reshape the way thousands of health issues are treated, the institute stresses. (OHSU/Kristyna Wentz-Graff). March 11, 2020 - Oregon Health & Science University (OHSU) held the first-ever gene therapy clinical trial, BRILLIANCE, to address blindness-causing gene mutation, LCA10, according to a recent press release. The Chinese government fears that he will not be the last person to perform criminal behavior using the new gene editing tool. Staff at Oregon Health & Science University's Casey Eye Institute perform the first in vivo CRISPR gene edit procedure for the BRILLIANCE clinical trial. READ MORE: New Ruling to Expand Public Access to Clinical Trial Data. The study will assess safety, tolerability, and efficacy in approximately 18 patients. Two separate Brilliance device sensors will be attached via standard adhesive pads to the left and right pectoralis major on each participant. BRILLIANCE uses AGN-151587, an experimental medicine delivered via sub-retinal injection under development for the treatment of Leber congenital amaurosis 10, a rare form of inherited blindness, or LCA10 and CEP290-related retinal dystrophy, according to the press release.