Sickle Cell in Focus Conference 2018

After an exciting event in Kingston, Jamaica in October 2017, we are thrilled to announce that the 12th annual Sickle Cell in Focus (SCiF) conference will return to the National Institutes of Health (NIH) on October 22-23, 2018.

SCiF is a two-day, intensive, educational update on sickle cell disease. This year’s conference will focus on management of the older adult patients, curative therapies, systems medicine, and will also include 2 debates on issues for which there are no clear answers.

This comprehensive and global exploration of the current medical trends and research results in sickle cell disease and thalassemia is geared towards consultants, trainees, doctors, healthcare professionals, and academics interested in hemoglobin disorders.

 

Date:

October 22-23, 2018

 

Location:

Natcher Conference Center, Ruth L. Kirschstein Auditorium

45 Center Dr.

National Institute of Health, Bethesda, Maryland, United States

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Preclinical Data on Proprietary AKT Inhibitor, ARQ 092, Demonstrating Effectiveness in the Treatment of Sickle Cell Disease Presented at the American Society of Hematology Annual Meeting

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.
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Acetylon Presents Preclinical Data Demonstrating the Utility of Selective HDAC1,2 Inhibition by ACY-957 to Induce Gamma Globin (HBG) Protein Expression for the Treatment of Sickle Cell Disease and Beta-Thalassemia

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.
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GlycoMimetics Announces Pricing of Public Offering of Common Stock

GlycoMimetics, Inc. (NASDAQ: GLYC), a clinical stage biotechnology company focused on the discovery and development of novel glycomimetic drugs, today announced the pricing of its underwritten public offering of 7,000,000 shares of its common stock at a price to the public of $11.50 per share. The gross proceeds from the offering are expected to be $80.5 million, before deducting underwriting discounts and commissions and estimated offering expenses payable by GlycoMimetics. The offering is expected to close on or about May 30, 2017, subject to customary closing conditions. Jefferies LLC and Cowen are acting as joint book-running managers for the offering. SunTrust Robinson Humphrey, Inc. is acting as lead manager for the offering. GlycoMimetics has granted to the underwriters a 30-day option to purchase up to 1,050,000 additional shares of common stock at the public offering price, less the underwriting discount.
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Rare patients with sickle cell disease live nearly twice national average

With a national median life expectancy of 42-47 years, people with sickle cell disease (SCD) face many challenges, including severe pain episodes, stroke, and organ damage. Compounding these complications is that SCD—an inherited, lifelong blood disorder characterized by rigid and sickle-shaped red blood cells that stick to the blood vessels, blocking blood flow—has only one FDA-approved treatment, no widely available cure, and some people may have limited access to appropriate care. However, a new report published online today in Blood, the Journal of the American Society of Hematology (ASH), shows that some people with mildly symptomatic SCD may live long lives with proper management of the disease, including strong family support and strict adherence to medication and appointments. This analysis of four case studies details the outcomes of four women with milder forms of SCD who have far surpassed the U.S. median of 47 years old for women with the disease, instead living as long as 86 years.
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medicalxpress.com
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medicalxpress.com
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Novartis acquires US pharma research firm Selexys Pharmaceuticals

Swiss-based pharmaceuticals giant Novartis said Monday it was acquiring Selexys Pharmaceuticals Corp., a US research lab in blood and inflammatory disorders, in a deal costing up to $665 million (627 million euros). Selexys specialises in sickle cell disease, an inherited form of anaemia that is most common among people of African descent. In a statement, Novartis said it exercised rights to buy the company after seeing results from a large trial into a drug to treat pain for people with the disease.
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Sickle cell cure is real, as this Kansas patient proves

Intense pain. Fatigue. Repeated infections, emergency room visits and hospitalizations.

Desiree Ramirez endured them often — until she became the first adult cured at a Kansas hospital of sickle cell disease.

Bone marrow stem cells donated by a stranger rescued Ramirez at age 23. Now, a year past transplant, with follow-up doctor’s office visits slowly receding, she finds herself eagerly anticipating a normal life, one without the inherited blood disorder that affects 70,000 to 100,000 Americans, mostly people of African heritage.

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stemcelltherapy.me
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stemcelltherapy.me
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Young Sickle Cell Patients Who Don’t Take Medication Have Lower Quality of Life

Adolescents and young adults with sickle cell disease (SCD) who don’t regularly take the medication hydroxyurea (or who don’t adhere to prescribed instructions) are generally those with medication-taking barriers such as forgetfulness, lack of access, poor medication knowledge, fear of side effects, or doubts about the drug’s treatment, according to new research. Patients with more barriers to taking their medication tended to have a lower quality of life.

The study, “Barriers to hydroxyurea adherence and health-related quality of life in adolescents and young adults with sickle cell disease,” was published online in the European Journal of Haematology. The researchers focused on clarifying the relationship between these barriers and quality of life for SCD patients.

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sicklecellanemianews.com
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sicklecellanemianews.com
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bluebird bio Announces Publication of Case Study on First Patient with Severe Sickle Cell Disease Treated with Gene Therapy in The New England Journal of Medicine

Patient treated with LentiGlobinTM drug product demonstrates high levels (~50% of total hemoglobin) of anti-sickling hemoglobin (HbAT87Q) 15 months after treatment. 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, today announced the publication in the New England Journal of Medicine of a case study on Patient 1204, the first patient with severe sickle cell disease (SCD) to be treated with gene therapy. This patient, who was 13 years old at the time of treatment, was treated with LentiGlobin drug product in the HGB-205 clinical study conducted in Necker Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France. The data in the publication reflect 15 months of follow-up, and a brief summary of this patient’s outcomes with 21 months of follow-up was presented at the 58th American Society of Hematology Annual Meeting in December 2016.
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SPCI Launched the SCOT (Sickle Cell Omega-3 Treatment) Trial in the United States with an Investigator Meeting Held December 16-17 in Atlanta

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.
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