News

January 21, 2020January 21, 2020

FT-4202, FORMA’s Experimental SCD Therapy, Fares Well in Phase 1 Trial of Healthy Volunteers

News

FT-4202, FORMA Therapeutics’ experimental disease-modifying therapy for sickle cell disease (SCD), shows a favorable safety and pharmacokinetic profile in healthy volunteers, according to data from a Phase 1 clinical ... Read more

January 16, 2020January 21, 2020

Sickle Cell Patient Describes ‘Rebirth’ After Gene Therapy

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For most of his 39 years, Charles Hough lived with extreme pain caused by sickle cell disease, which dramatically reduced blood flow throughout his body. But thanks to the ... Read more

January 14, 2020January 14, 2020

Emmaus Partners with US Bioservices to Offer SCD Patients New Treatment Adherence Program

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To help empower individuals living with sickle cell disease (SCD), biopharmaceutical company Emmaus Life Sciences has teamed up with national specialty pharmacy US Bioservices on a treatment adherence program ... Read more

January 9, 2020January 9, 2020

FDA Grants Rare Pediatric Disease Status to Investigational Gene Therapy ARU-1801 for Sickle Cell

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The U.S. Food and Drug Administration (FDA) granted rare pediatric disease designation to ARU-1801, Aruvant’s experimental gene therapy for the treatment of sickle cell disease (SCD) and beta-thalassemia. This ... Read more

January 7, 2020January 7, 2020

GBT and Syros Partner to Develop Sickle Cell Treatments That Boost Fetal Hemoglobin

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Global Blood Therapeutics (GBT) and Syros Pharmaceuticals have teamed up to discover, develop, and market new therapies for sickle cell disease (SCD) and beta thalassemia. Under the collaboration agreement, ... Read more

January 2, 2020January 3, 2020

NIH Grant Backs Project to Develop New Pain Therapy for SCD Patients

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The National Institutes of Health (NIH) has awarded a grant to researchers at Carnegie Mellon University’s College of Engineering to support their project to develop a non-invasive, non-toxic and non-addictive pain ... Read more

December 17, 2019December 17, 2019

California Earmarks $15M for Expanding Care of Adult SCD Patients

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California’s legislature has committed $15 million toward sweeping enhancement of adult sickle cell disease (SCD) care in a state with historically underserved patients. The initiative is intended to improve ... Read more

December 12, 2019December 12, 2019

Gene Therapy LentiGlobin Continues to Show Promising Results, Phase 1/2 Trial Data Shows

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LentiGlobin, Bluebird Bio’s investigational gene therapy for sickle cell disease (SCD), continues to show promising results in SCD patients participating in the company’s Phase 1/2 HGB-206 clinical trial, according ... Read more

December 10, 2019December 10, 2019

Gene Therapy Takes Center Stage at 2019 NORD Summit

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With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough ... Read more