Online Exclusives

Online Exclusives

Cellectis Relaunches Halted CAR-T Trials

The U.S. Food and Drug Administration lifted the clinical hold on two phase I trials of Cellectis’ UCART123, its gene-edited allogeneic chimeric antigen receptor...

Access to Care, Survival Are Enduring Hurdles for Patients With Sickle Cell Disease

As part of an ongoing series on discrimination in the United States, NPR reported that while life expectancy for almost every major illness is...

Trump Administration to Support Work Requirements for Medicaid Beneficiaries

The Trump Administration announced it will approve proposals from states to require work or community engagement for those who want to receive Medicaid. Community...

FDA Approves First-Ever Treatment for Rare Blood Cancer

The U.S. Food and Drug Administration (FDA) expanded the approval of vemurafenib, a kinase inhibitor, to include the treatment of adult patients with BRAF-V600-mutated...

Congress Considers Eliminating Incentives for Orphan Drugs in New Tax Bill

The Republican tax plan includes a proposal to end tax credits for the development of drugs for rare diseases. Ending the credit, which is...

FDA Grants Accelerated Approval to Acalabrutinib for Mantle Cell Lymphoma

The U.S. Food and Drug Administration (FDA) granted accelerated approval to acalabrutinib, a second-generation Bruton tyrosine kinase, as secondline treatment for patients with mantle...

Medical Device and Drug Shortages Loom During Puerto Rico’s Hurricane Recovery

When Hurricane Irma devastated Puerto Rico, the more than 80 pharmaceutical manufacturing facilities and more than 50 medical-device factories in the U.S. territory were...

Early Data From Ongoing JCAR017 Trials Show High Remission Rates

Preliminary data from the ongoing TRANSCEND trial showed that patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who were treated with the...

Improvements in Manufacturing Overcomes Challenges of Gene Therapy for Sickle Cell Disease

Data from two patients in an ongoing phase I/II clinical trial of LentiGlobin BB305, a gene therapy for sickle cell disease (SCD), suggest that...

Enasidenib Manufacturers Plan to Submit Mutant IDH1 Inhibitor to FDA

Agios Pharmaceuticals announced plans to seek approval from the U.S. Food and Drug Administration (FDA) for its second leukemia drug – ivosidenib, a mutant IDH1...
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Current Issue

November 2017, Volume 3, Issue 13

This issue features a behind-the-scenes look at the ASH annual meeting, bringing newly approved gene therapies to patients, and more.