News

US Patient Groups Give Thumbs-Up to Rule Against Surprise Billing

The National Organization for Rare Disorders (NORD) is applauding the Biden administration for announcing a rule to protect consumers from surprise medical billing, in a joint statement with 26 other U.S. patient organizations. The interim final rule will implement patient protections required by the No Surprises Act. Surprise…

UCSF Awarded Nearly $8.4M for CRISPR_SCD001 Gene Editing Trial

The University of California, San Francisco (UCSF) has been awarded $8.39 million to fund a Phase 1/2 clinical trial of CRISPR_SCD001, the first non-viral and CRISPR-Cas9-based gene editing therapy for sickle cell disease (SCD). Researchers will use the gene-editing technology to attempt to correct a genetic mutation in the blood…

Partnership Aims to Lower Out-of-Pocket Costs for Rare Disease Meds

AllianceRx Walgreens Prime, a specialty and home delivery pharmacy, is partnering with TailorMed, a healthcare technology company, to help lower out-of-pocket prescription costs for specialty pharmacy patients. Medications attained through specialty pharmacies are those used to treat rare and chronic conditions in the U.S., and are often extremely costly. For…

Emmaus Seeks Approval of Oral Medication Endari in Kuwait

Emmaus Life Sciences has submitted an application seeking the approval of its oral medication Endari (L-glutamine) to treat sickle cell disease (SCD) in Kuwait. The company announced that the Kuwait Drug and Food Control administration, which is responsible for registering pharmaceutical products in the country, has accepted…

Nominations Open for 2022 Eurordis Black Pearl Awards

Nominations are now open for the worldwide 2022 Black Pearl Awards from Eurordis-Rare Diseases Europe. The 12 award categories recognize individual advocates, policy makers, researchers, organizations, and companies who work to make a difference for the global rare disease community. The deadline for nominations is Sept. 10…

‘Cheek Week’ Seeks Donors to Join Marrow Donor Program

As a stem cell transplant is the only curative treatment for sickle cell disease (SCD), a blood disorder that disproportionately affects Blacks and African Americans, “Cheek Week” is again calling for the participation and inclusion of donors from different ethnic backgrounds in the Be The Match registry,…

Study to Evaluate Using JSP191 With ARU-1801 as Gene Therapy

A new research collaboration between Jasper Therapeutics and Aruvant Sciences will evaluate the use of JSP191, Jasper’s anti-CD117 monoclonal antibody, as a conditioning agent for ARU-1801, Aruvant’s experimental gene therapy for sickle cell disease (SCD). “The unique attributes of ARU-1801 enable us to bring a potentially curative one-time therapy…

CTX001 Continues to Show Promise in Severe SCD

A single dose of CTX001, an experimental gene-editing cell therapy, rapidly increases the levels of hemoglobin and prevents vaso-occlusive crises (VOCs) for up to nearly two years in people with severe sickle cell disease (SCD), according to interim data from the Phase 1/2 CLIMB-SCD-121 clinical trial.