Sancilio Pharmaceuticals Company, Inc. (SPCI) today announced the start of the first of two placebo controlled clinical trials in US pediatric patients affected with Sickle Cell Disease (SCD). Recruitment in the trial is underway and enrollment is expected to be completed by Q2 2017 and will be immediately followed by a second placebo controlled clinical study in Q3 2017. SCD is estimated to affect 100,000 Americans, with a disproportional impact on African Americans. “It has taken us nearly 3 years to get to the point of dosing children in the United States who have this horrible disease. I am excited by the data we’ve seen in studies performed abroad, but having only children from the United States in such a study will be exciting and conclusive,” said Dr. Frederick Sancilio, CEO of SPCI.

First patient with severe sickle cell disease treated with gene therapy remains free of clinical symptoms 21 months after receiving LentiGlobin Drug Product. Ongoing transfusion independence and sustained production of HbAT87Q in patients with transfusion-dependent β-thalassemia. Company to host event at ASH with live webcast, Monday, December 5 at 8:30 p.m. PT. bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, announced the presentation of new data from the ongoing HGB-205 clinical study evaluating its LentiGlobin product candidate in patients with transfusion-dependent β-thalassemia (TDT) and severe sickle cell disease (SCD) at the 58th American Society of Hematology Annual Meeting.

Acetylon Pharmaceuticals, Inc., the leader in the development of selective histone deacetylase (HDAC) inhibitors for enhanced therapeutic outcomes, today announced that it will present preclinical data comparing the effects of alternative dosing schedules for the selective HDAC1,2 inhibitor, ACY-957, on gamma globin (HBG) protein expression. In an oral presentation at the American Society of Hematology Annual Meeting (ASH) in San Diego, California, Jeffrey R. Shearstone, Ph.D., Associate Director of Biology at Acetylon, will present data demonstrating that treatment with ACY-957 was well tolerated in a 3-week dosing regime and led to greater than 50-fold increases of HBG mRNA and protein in non-anemic primates.

ArQule, Inc. (Nasdaq: ARQL) today announced that preclinical data was presented on its proprietary AKT inhibitor, ARQ 092, in an oral presentation by the University of Illinois College of Medicine at the American Society of Hematology (ASH) Annual Meeting. The presentation highlighted preclinical studies of ARQ 092 in sickle cell disease (SCD). ARQ 092 is an orally available, selective pan-AKT inhibitor.

The NHLBI Annual Sickle Cell Disease Clinical Research Meetings provide a yearly forum for investigators, practitioners, and interested health care providers to discuss the progress of ongoing clinical trials, hear presentations about new developments in scientific and clinical aspects of SCD, and interact with other investigators and NHLBI Program Staff. This year, the meeting will begin on August 15, 2016, and end on August 17, 2016. There will be three symposiums, one on each day: Monday, August 15, 2016: Symposium: Stem Cell Transplantation in Sickle Cell Disease (Lakshmanan Krishnamurti, MD, Emory, organizer). Tuesday, August 16, 2016: Symposium for Data Science for Sickle Cell Disease and Health Care (Ellen Werner, PhD, NHLBI). This symposium will serve as a practical introduction to the data science for clinicians and health care providers. Wednesday, August 17, 2016: Symposium on Transfusion Medicine in Sickle Cell Disease (Ify Osunkwo, MD (University of North Carolina, Charlotte) and Julie Kanter, MD (Medical University of South Carolina, organizers). This meeting is an opportunity to meet and exchange ideas with a diverse group of SCD investigators and providers, as well as NIH Program Officers in an informal setting.

The Sickle Cell Community Consortium is pleased to announce the 2017 Leadership Summit & General Assembly of Advocates and Community-based organizations (CBOs). The Consortium is a 501(c)(3) not-for-profit collective of Sickle Cell Community-Based Organizations (CBOs), Community Partners, Sponsored Patient Organizations, Patient/Caregiver Advocates and Healthcare/Research Advisors- each dedicated to sickle cell advocacy, education, research, community awareness and patient support throughout the country. The Consortium was created to provide an organizing arm to enable our communities to identify and address local and national needs. Patterned after the United Nations, and following a model of Collective Impact, we provide a platform to “amplify the power of the patient voice”. As part of this collaborative effort, we are excited to host the 2017 Leadership Summit and General Assembly, March 16-19, 2017 in Atlanta, Georgia. This meeting is open to the public. You are invited to join us as we continue to effect patient-powered change in our community.

A blood test Thursday revealed that Arizona Cardinals wide receiver John Brown carries the sickle-cell trait and has been listed as doubtful for Sunday's game, coach Bruce Arians said Friday. Brown experienced leg pain before Monday night's win over the New York Jets and was diagnosed with sore hamstrings after the game. But an MRI this week didn't reveal any issues with his hamstrings, which led to the blood test. "We're investigating on how to help him get better as fast as possible," Arians said.

Highlights · The global apheresis equipment market is expected to reach USD 3.7 billion by 2024, according to a new report by Grand View Research, Inc. · The apheresis equipment industry is expected to be impacted by factors such as rising demand for plasma, platelets and other blood components, increasing incidence of chronic illnesses, presence and development of ideal reimbursement policies across various regions, and supportive government initiatives. · Growing incidence of blood related disorders leading to the rise in demand for platelets and plasma is expected to serve the market as a high impact rendering driver. · Disposable apheresis kits are anticipated to dominate the product segment by 2024. · The photopheresis procedure is anticipated to witness lucrative growth owing to its rising demand in treatment.

Research team establishes biomarkers for the inherited blood disorder. Researchers have found new biophysical markers that could help improve the understanding of treatments for sickle cell disease, a step toward developing better methods for treating the inherited blood disorder that affects an estimated 80,000 to 100,000 Americans each year.