Clinical TrialsInformation and insight on the latest clinical trials
- FDA Grants CTX001 Fast Track Status for Sickle Cell Disease
The U.S. Food and Drug Administration (FDA) has granted fast track status to investigational therapy CTX001 to treat sickle cell disease.
- Sickle Cell Disease Community Hopeful that Genome Editing Will Rechart Course of the Disease
In the first study of its kind, researchers explored patients’, parents’ and physicians’ perspectives on the use of CRISPR-Cas9 gene-editing to reverse sickle cell disease (SCD). CRISPR-Cas9 is a gene editing technology that researchers have harnessed …
- Novel Approach to Gene Therapy for Sickle Cell Disease Shows Promise
Erica B. Esrick, MD, Hematologist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center reported on her pilot trial in which one adult patient with Sickle Cell disease (SCD) received an infusion of his own stem cells in which a genetic “switch” was flipped on to induce the cells to both start producing healthy hemoglobin and stop producing unhealthy “sickle” hemoglobin.
- Beat AML Trial: Rapid Genetic Screening in Acute Myeloid Leukemia
Amy Burd, PhD, Vice President of Research Strategy at the Leukemia and Lymphoma Society (LLS), discusses her group’s multiarm, multisite collaborative trial which aims to test targeted therapy approaches for improving the generally poor prognosis among patients with AML.
- Potential Use of Venetoclax in Multiple Myeloma
Rod Humerickhouse, MD, PhD, asset strategy leader at AbbVie discusses the potential use of venetoclax for the treatment of patients with multiple myeloma at the 60th ASH Annual Meeting in San Diego.
- Primary Haemophagocytic Lymphohistiocytosis: Overview and New Treatment Option
Carol Satler, MD Vice President of Medical Affairs in North America for Sobi, discusses the ultra rare disease primary haemophagocytic lymphohistiocytosis (HLH) and a new FDA approved treatment Emapalumab.
- CAR-T Cell Update: Therapy Improves Outcomes for Patients With B-cell Lymphoma
Richard Thomas Maziarz, MD discusses the JULIET trial, a phase-2 study of tisagenlecleucel (marketed as KYMRIAH).
- Complications from a Hematopoietic cell Transplantation
Dr. Jonathan Peled of Memorial Sloan Kettering Cancer Center discusses his study which looked at whether pre-HCT microbiota configuration may be an important determinant of post-transplantation outcomes.
- SOBI Overview at ASH: Data from Phase 2/3 Clinical Study of Emapalumab-lzsg in Primary HLH
Rami Levin, President of North America for Swedish Orphan Biovitrum (SOBI) discusses his company’s recent FDA approval, Sobi’s commitment to the rare disease community, and how Sobi is growing its presence in North America.
- Epstein-Barr Virus Associated Post-transplant Lymphoproliferative Disorder (EBV+ PTLD)
Mohamad Mohty, MD, PhD, discusses EBV+ PTLD is an aggressive lymphoma that occurs following bone marrow transplant (also known as hematopoietic stem cell transplant, or HCT) or solid organ transplant (SOT).