Picking up on the ongoing “Family Man” saga, in New York Court, EGT claims that Zynteglo, the Bluebird Bio product for Thalassemia, created Clonal Dominance. EGT alleges that Bluebird Bio assisted Memorial Sloan Kettering in orchestrating the sabotage of EGT’s product...
Sickle Cell Disease
Category
Rice’s Gang Bao Awarded $2.45m Grant from NHLBI for Gene Editing Study Targeting Sickle Cell Disease
A Rice University lab won a prestigious National Institutes of Health grant to pursue gene-editing research that they hope will help lead to a cure for sickle cell disease (SCD). A four-year R01 grant to Gang Bao, Rice’s Foyt Family Professor of Bioengineering and a...
Pfizer Rare Disease Lead on the Importance of Clinical Trials to Pursue Rare Disease Treatments
Dr. Kevin Williams recently elaborated on the importance of clinical trials. The chief medical officer for Rare Disease Pfizer Biopharmaceuticals described his disappointment in a Phase III clinical trial where rivipansel, a potential treatment for vaso-occlusive...
ASH Research Collaborative Data Hub Receives Shared Industry Datasets from Novartis & Amgen
The ASH Research Collaborative (ASH RC) has collaborated with two industry leaders, Novartis and Amgen, to include de-identified patient data from three separate studies of nearly 500 patients living with Sickle Cell Disease (SCD) and more than 1,000 patients with...
A Small Study Raises Big Hopes That Gene-Editing Can Treat Blood Diseases
A small study can raise big expectations—especially in the nascent industry of gene-editing to treat serious diseases. Such is the case for CTX001, a closely-watched therapy from upstart biotech CRISPR Therapeutics and its much larger partner, rare disease specialist...
Errant Gene Therapeutics Update: Tanzania, Court Appeals and More
TrialSite News is, above all, dedicated to transparency and access to clinical trials with an emphasis on clinical research sites and investigators—those who are on the frontlines of clinical research. We bring to light the events and highlights of the clinical...
Changing the Face of Clinical Trials
Judging the value of new drugs and medical devices is becoming an increasingly important issue with the proliferation of alternative treatment options. Beyond key aspects such as clinical efficacy and safety, other considerations such as quality of life and costs are...
Airway Inflammation and Lung Function in Sickle Cell Disease—The Study
A new study by De, Agrawal, Morrone et al, challenges the common notion that airway inflammation in Sickle Cell Disease (SCD) is secondary to asthma, even though the two disorders often coexist. In a recent pilot, cross sectional study, both systemic and airway...
Global Blood Therapeutics Reports Updated Data from Phase 3 HOPE Study of Voxelotor in Sickle Cell Disease
Global Blood Therapeutics reported new data from its Phase III HOPE trial of voxelotor in sickle cell disease (SCD), showing the primary endpoint, improvement in hemoglobin greater than 1 g/dL at 24 weeks, was reached. The research was published in The New England...
CHU Ste-Justine Evaluates Clinical Protocol Using Intranasal Fentanyl for Vaso-Occlusive Crisis in SCD Patients
Investigators from CHU Ste-Justine, Montreal Quebec, Canada noted vaso-occlusive crisis (VOC) is one of the most frequent causes of emergency visits and admissions in children with sickle cell disease (SCD). Their recent study aimed to evaluate whether the use of a...
CRISPR Therapeutics, Vertex Clinical Update on Gene-Editing Therapy CTXoo1
CRISPR Therapeutics (NASDAQ: CRSP) and Vertex Pharmaceuticals (NASDAQ: VRTX) report that recent progress in the clinical development programs for CTX001, an investigational autologous, CRISPR/Cas9 gene-edited hematopoietic stem cell therapy being evaluated for...
TrialWatch: REACH Trial Shows Sickle Cell Anemia Treatment Safely Lowers Disease Burden in African Children
The REACH trial is a prospective, phase I/II open-label dose escalation trial of hydroxyurea for children with confirmed SCA between 12 months and 10 years of age. The short-term goal was to obtain critical pilot data regarding the feasibility, safety and benefit of...
Indian Research Sites Including Tata Memorial Hospital Plan Large Cannabis Clinical Trial in India
Kalyan Ray of Deccan Herald writes that Indian researchers will spearhead that country’s first set of cannabis-centered clinical trials. The trials will be led by physicians at Tata Memorial Hospital in Mumbai, All India Institute of Medical Sciences in Delhi and the...
American Society of Hematology Plans Sickle Cell Disease Trial Network
As reported in MDLinx, the American Society of Hematology (ASH) is launching a sickle cell disease (SCD) clinical trials network in order to accelerate the development of new therapies for a patient community that has very few treatments and curative options. The core...
UCSF Stem Cell Research & Mark Benoif Grant a Powerful Force in Fighting Sickle Cell Disease
UCSF Benoif Children's Hospital Oakland Blood and Marrow Transplantation Program shoots for a Moon shot to cure Sickle Cell Disease. Over 30,000 infants each year are born with the genetic disease. The first genetic disease to be discovered, researchers at prestigious...