Centers for Medicare & Medicaid Services (CMS) Administrator Chiquita Brooks-LaSure to deliver keynote
SOUTH SAN FRANCISCO, Calif., and BALTIMORE – September 8, 2021 – Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) and the Sickle Cell Disease Association of America, Inc. (SCDAA) will host the 10^th Annual Sickle Cell Disease (SCD) Therapeutics Conference on Wednesday, September 15th. The virtual conference, which takes place during National Sickle Cell Awareness Month, is open to the public and will be livestreamed on the SCD Therapeutics Conference website and the Facebook page of Sickle Cell Warriors, an SCD community organization.
Chiquita Brooks-LaSure, Administrator for the Centers for Medicare & Medicaid Services (CMS) of the U.S. Department of Health and Human Services, will deliver the keynote address. A former policy official who played a key role in guiding the Affordable Care Act (ACA) through passage and implementation, Ms. Brooks-LaSure has decades of experience in the federal government, on Capitol Hill, and in the private sector.
“Progress in the treatment of sickle cell disease in recent years has provided hope and optimism for the future. But significant gaps in access to high quality care and other barriers continue to plague this community,” said Kim Smith-Whitley, M.D., executive vice president and head of research and development at GBT. “With this annual meeting now in its 10^th year, GBT is pleased to once again partner with the SCDAA and Sickle Cell Warriors to make this year’s conference possible – especially given the dramatic impact that the COVID-19 pandemic has had on people affected by SCD. We are inspired to convene such a diverse, passionate group of researchers, clinicians, patients, advocacy leaders, policy makers, industry representatives and others to address the complex challenges this community faces.”
The event will also feature sessions on a variety of critical topics, including the impact of the novel COVID-19 vaccines and the experiences of the SCD community with these vaccines.
The program will include panel discussions featuring leading physicians from around the world, patients, advocates, and policy makers, as well as updates from GBT’s 2021 Access to Excellent Care for Sickle Cell Patients (ACCEL) Grant recipients, who are focused on delivering high-quality healthcare to people living with SCD.
“In the past year, the COVID-19 pandemic has magnified the long-standing inequities faced by the sickle cell community and impacted the daily lives of so many people living with SCD. We are privileged to unite leaders from around the world on important and timely topics and create change for the better.” said Beverley Francis-Gibson, M.A., president and CEO of the SCDAA. “The conference will provide a much-needed forum for the broader SCD community to come together and discuss the urgent needs, latest advancements, and future possibilities as we imagine a better world for people with sickle cell disease.”
The 10^th Annual SCD Therapeutics Conference will feature the following speakers and panelists:

• Lewis Hsu, M.D., Ph.D., Chief Medical Officer, Director of the Sickle Cell Center and Professor of Pediatrics; Pediatric Hematologist, University of Illinois at Chicago
• Phyllis Arthur, M.B.A, Vice President, Infectious Diseases and Emerging Science Policy; Biotechnology Innovation Organization (BIO)
• Professor Baba Inusa, M.D., Lead Consultant Paediatric Sickle Cell and Thalassaemia, Evelina London Children’s Hospital; Guy’s and St Thomas NHS Trust, Women and Children’s Health; Faculty of Life Sciences & Medicine, King’s College London
• Clarisse Lopes de Castro Lobo, M.D., Instituto de Hematologia Arthur de Siqueira Cavalcanti (HEMORIO)
• Caterina P. Minniti, M.D., Professor, Departments of Medicine and Pediatrics, Albert Einstein College of Medicine
• Immacolata Tartaglione, M.D., Ph.D., Department of Woman, Child and General and Specialist Surgery, Università degli Studi della Campania “Luigi Vanvitelli”
• Derek Robertson, President, Maryland Sickle Cell Disease Association
• James Griffin, Speaker, Advocate & Author of Breaking Silence: Living With Sickle Cell Anemia
• Shanetta Richardson, Patient Advocate, US
• Chanel Taylor, Patient Advocate & Founder, Unsickle My Cells, UK
• Carolyn Rowley, Ph.D., Executive Director, Cayenne Wellness Center
• Emma Andelson, M.P.A., Program Manager, Sick Cells
• Lakiea Bailey, Ph.D., Executive Director, Sickle Cell Community Consortium
• Velvet Brown-Watts, M.S.W., Executive Director, Supporters of Families with Sickle Cell Disease
• Reginald French, Chief Executive Officer, The Sickle Cell Foundation of Tennessee

Multiple companies that are developing treatments for patients with SCD will present at the conference. They include Agios Pharmaceuticals, Bluebird Bio, Forma Therapeutics, GBT, Imara, Inc., and Vertex Pharmaceuticals/CRISPR Therapeutics.
For more information about the conference or to register to attend, visit http://www.scdconference.com.  Follow the conversation on Twitter using the hashtag #SCDTC.
About Sickle Cell Disease
Sickle cell disease (SCD) affects an estimated 100,000 people in the United States,¹ an estimated 52,000 people in Europe,² and millions of people throughout the world, particularly among those whose ancestors are from sub-Saharan Africa.¹ It also affects people of Hispanic, South Asian, Southern European and Middle Eastern ancestry.¹ SCD is a lifelong inherited rare blood disorder that impacts hemoglobin, a protein carried by red blood cells that delivers oxygen to tissues and organs throughout the body.³ Due to a genetic mutation, individuals with SCD form abnormal hemoglobin known as sickle hemoglobin. Through a process called hemoglobin polymerization, red blood cells become sickled – deoxygenated, crescent-shaped and rigid.^3-5 The sickling process causes hemolytic anemia (low hemoglobin due to red blood cell destruction) and blockages in capillaries and small blood vessels, which impede the flow of blood and oxygen throughout the body. The diminished oxygen delivery to tissues and organs can lead to life-threatening complications, including stroke and irreversible organ damage.^4-7
 
About Global Blood Therapeutics
Global Blood Therapeutics (GBT) is a biopharmaceutical company dedicated to the discovery, development and delivery of life-changing treatments that provide hope to underserved patient communities. Founded in 2011, GBT is delivering on its goal to transform the treatment and care of sickle cell disease (SCD), a lifelong, devastating inherited blood disorder. The company has introduced Oxbryta^® (voxelotor) tablets, the first FDA-approved treatment that directly inhibits sickle hemoglobin polymerization, the root cause of red blood cell sickling in SCD. GBT is also advancing its pipeline program in SCD with inclacumab, a P-selectin inhibitor in Phase 3 development to address pain crises associated with the disease, and GBT021601 (GBT601), the company’s next-generation hemoglobin S polymerization inhibitor. In addition, GBT’s drug discovery teams are working on new targets to develop the next wave of potential treatments for SCD. To learn more, please visit www.gbt.com and follow the company on Twitter @GBT_news.
About SCDAA
SCDAA’s mission is: To advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure. Visit www.sicklecelldisease.org.
References

1. Centers for Disease Control and Prevention website. Sickle Cell Disease (SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html. Accessed June 3, 2019.

1. European Medicines Agency. https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125. Accessed June 12, 2020.
2. National Heart, Lung, and Blood Institute website. Sickle Cell Disease. https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease. Accessed August 5, 2019.
3. Rees DC, et al. Lancet. 2010;376(9757):2018-2031.
4. Kato GJ, et al. Nat Rev Dis Primers. 2018;4:18010.
5. Kato GJ, et al. J Clin Invest. 2017;127(3):750-760.
6. Caboot JB, et al. Paediatr Respir Rev. 2014;15(1):17-23.

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Contact:
Steven Immergut (media)
650.410.3258
simmergut@gbt.com
Courtney Roberts (investors)
650.351.7881
croberts@gbt.com
SCDAA Contact Information:
Emma Day
eday@sicklecelldisease.org
Kyri Jacobs
kjacobs@sicklecelldisease.org
 
   

Sickle Cell Disease Association of America named John Otsuki as government relations manager. Otsuki brings experience in regulatory and legislative affairs, government relations and strategic planning.
Prior to joining the Sickle Cell Disease Association of America, Otsuki was a government relations manager at Jones Walker LLP, where he maintained relationships with members of Congress and government agencies, executed regulatory processes, developed protocols and prepared reports on regulatory and legislative affairs. He previously served as a legislative analyst and research and administrative assistant at the law firm and as a legislative intern for a member of Congress.
Otsuki holds a Bachelor of Arts in political science from Auburn University.  

Sickle Cell Disease Association of America (SCDAA) named 14-year-old Ayana Lee Johnson the 2021-2023 SCDAA National Teen Ambassador. She was selected through a nationwide competition formerly known as the Poster Child Contest that started in 1976. Her reign will begin with an official coronation during SCDAA’s Annual National Convention Oct. 12-16.

In her role as SCDAA National Teen Ambassador, Johnson will make public appearances and act as an emissary, assisting with education campaigns and securing public and private sector support for the SCDAA mission. She will serve as a goodwill ambassador for SCDAA and be a positive role model for teens and other individuals living with sickle cell disease.

“SCDAA is thrilled with the selection of Ayana as our new Teen Ambassador,” said SCDAA President and CEO Beverley Francis-Gibson. “Her determination to fight for other SCD warriors and raise awareness of what it is like to live with sickle cell disease to anyone who will listen, along with her sparkling personality, are just a few reasons why she was chosen. She is resilient and determined not to be limited or defined by SCD.”

Johnson is an honors student at Nansemond River High School in Suffolk, Virginia, and she attends the Governor’s School of the Arts. She is a competitive dancer with many national titles and has been accepted into summer intensives with both the Dance Theatre of Harlem and the Joffrey Ballet School. Johnson also plays violin and competes in pageants. She currently holds the title of Miss America’s Outstanding Teen Piedmont Region 2020-2021.

“Sickle cell disease does not define me,” said Johnson. “I am a warrior living my best life.”

Johnson was featured in Suffolk News Herald’s 20 Under 21, and among her many accomplishments, she is the recipient of the St. Peter Claver Service and Sickle Cell Activism Award; the Making the Difference – Sickle Cell Champion Warrior Award and Scholarship; and the 2019 Excellence Girls Club Pioneer Award. In 2020-2021, she completed 200 volunteer hours, some of them working as an American Red Cross Blood Drive escort and greeter.  

New campaign to raise awareness about connection between sickle cell trait and deadly form of kidney cancer.

The Kidney Cancer Association (KCA) and the Sickle Cell Disease Association of America, Inc. (SCDAA) are partnering to launch the “KNOW & TELL” initiative today to raise awareness about sickle cell trait (SCT) and its link to a rare, aggressive type of kidney cancer called renal medullary carcinoma (RMC).
The year-long initiative seeks to promote the early identification of RMC by encouraging people to know their SCT status and inform their family and health care providers about the connection between SCT and RMC. Being aware of both SCT status and the rare RMC symptoms could help in identifying RMC early, obtaining appropriate care and increasing positive outcomes.
RMC typically affects young Black men under age 30 and about half of those diagnosed do not survive beyond 13 months.¹ RMC makes up less than 1% of all kidney cancers but is almost exclusively found in people who carry SCT, a usually benign condition that can occasionally cause symptoms similar to sickle cell anemia.
“One of my key platforms is bringing greater awareness to rare types of kidney cancer and supporting research into treatments for underrepresented populations,” said the KCA’s Medical Director Sallie McAdoo, MS, CGC. “We’re proud to partner with SCDAA to bring greater awareness to RMC and its association with SCT through KNOW & TELL in the hope of helping those who are at risk identify it sooner, increase research into the condition, and improve outcomes.”
The SCDAA is an advocacy organization focused on education and advancing research for sickle cell conditions.
“We are excited to be partnering on this vital strategic initiative to increase awareness about SCT and one of the many possible complications tied to the trait,” said Beverley Francis-Gibson, president and CEO of the Sickle Cell Disease Association of America. “This campaign shows that knowledge is power and can have a big impact on care.”
Newborns are routinely screened for SCT however, those who carry the trait may not show symptoms and are therefore often not told they have it. Low awareness about SCT status and the lack of understanding about the link between SCT and RMC contribute to late-stage RMC diagnoses and shortened life expectancy.
“The RMC community may be smaller but is no less important than that of other cancer types and they are passionate about advocating for greater awareness about the disease and access to clinical trials for the opportunity to better understand and treat this rare cancer,” said Gretchen E. Vaughan, KCA’s president and CEO. “Partnering with the SCDAA for this initiative is the first step toward bridging the knowledge gap about the link between RMC and SCT.”
KNOW & TELL launches on June 18, following World Kidney Cancer Day on June 17, and in advance of World Sickle Cell Day on June 19.  Learn more at www.kidneycancer.org/know-and-tell.

1. National Association for Rare Disorders

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About the Kidney Cancer Association
The Kidney Cancer Association is a global community dedicated to serving and empowering patients and caregivers, and leading change through advocacy, research, and education in order to be the universal leader in finding the cure for kidney cancer. Founded in 1990 by Eugene P. Schonfeld and a small group of patients and doctors in Chicago, Illinois, the KCA has grown into an international non-profit organization based in Houston, Texas. The KCA promotes scientific advances through two annual research symposiums and a robust grant program, participates in legislative advocacy, and seeks to be a source of education and resources for patients, caregivers, and anyone impacted by kidney cancer.
Contact: Radha Chitale, Director of Communications. 847.332.1051 ext. 113 | rchitale@kidneycancer.org
About the Sickle Cell Disease Association of America
Sickle Cell Disease Association of America advocates for people affected by sickle cell conditions and empowers community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure. The association and more than 50 member organizations support sickle cell research, public and professional health education and patient and community services. www.sicklecelldisease.org  

Download PDF | Visit the GBT Website  

Sickle Cell Disease Association of America partnered with the Sickle Cell Community Consortium to advocate for legislation benefiting people with sickle cell disease and their families. The partnership includes collaboration on the association’s annual advocacy day initiatives, meetings and trainings and in developing federal legislative priorities.
“Sickle Cell Disease Association of America and the Sickle Cell Community Consortium share the same mission of improving the lives of people with sickle cell disease,” said Beverley Francis-Gibson, president and CEO of the Sickle Cell Disease Association. “We’re excited to work with the consortium, which brings a range of organizations, advocates and advisers that will help us achieve our legislative goals together.”
A nonprofit formed in 2014, the Sickle Cell Community Consortium consists of sickle cell community organizations, patient and caregiver advocates, community partners and medical and research advisers working together to represent people with sickle cell disease. The consortium identifies and implements strategies and partnerships to address needs in the sickle cell community.
Sickle Cell Disease Association of America’s annual advocacy day, supported by the partnership between the association and consortium, generates public awareness of sickle cell disease and kickstarts momentum to push for legislative reform. The day provides training, resources and guidance to participants interested in advocacy work.  

Sickle Cell Disease Association of America, Inc. (SCDAA) and Hemanext Inc., a privately held medical technology company dedicated to improving the quality, safety, efficacy and cost of red blood cell (RBC) transfusion therapy, today announced the launch of new educational material to help SCDAA deliver on its mission and meet its goals. Hemanext has sponsored the creation of a set of educational materials, one for a child and one for a caregiver, to educate on blood transfusions. This collaboration is part of SCDAA and Hemanext’s partnership, which began in 2020.
Sickle cell disease (SCD) affects 100,000 individuals in the United States, disproportionately affecting African Americans. SCD occurs in about one in 365 Black or African American births.¹ The genetic disease is associated with serious, life-threatening complications, including stroke and acute chest syndrome (ACS).^2,3 As result, people with SCD often require chronic red blood cell transfusions,² which for some patients is a life-saving therapy^.3
Children and parents may experience anxiety because they are unaware of the process to receive a blood transfusion. The goal of these two educational materials is to inform and empower patients and their caregivers about this important therapy. SCDAA will make these the resources available to the sickle cell community.
“During a review of our currently available educational resources, we identified the opportunity to help young sickle cell warriors prepare for their transfusions,” said SCDAA President and CEO Beverley Francis-Gibson. “We appreciate that Hemanext has stepped up to help us fill this information need and make a difference breaking the sickle cycle.”
“It is a privilege to continue our partnership with Ms. Francis-Gibson and her dedicated team at SCDAA, the premier sickle cell organization,” said Hemanext President and CEO Martin Cannon. “We are committed to helping SCDAA achieve its mission and enhance the lives of members of the sickle cell community.”
“As we enter the second year of our alliance, we will continue to look for ways to support SCDAA, patients, and families during these difficult times,” said Alex Marichal, VP, Marketing, Hemanext. 
ABOUT SCDAA
SCDAA’s mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure. Visit www.sicklecelldisease.org.
ABOUT HEMANEXT
Hemanext’s mission is to help patients enjoy healthier lives through safer transfusions. Hemanext’s technology is a processing and storage system that is designed to remove the fuel for oxidative damage to red blood cells. Hemanext is focused on supporting clinicians and healthcare practitioners who prescribe life-saving RBC transfusions to their patients. Visit Hemanext.com to learn more.

1. Data & Statistics on Sickle Cell Disease. Centers for Disease Control and Prevention. https://www.cdc.gov/ncbddd/sicklecell/data.html. Accessed March 19, 2021.
2. Understanding Sickle Cell Disease. American Society of Hematology, 2019. Available at https://www.hematology.org/education/clinicians/guidelines-and-quality-care/clinical-practice-guidelines/sickle-cell-disease-guidelines. Accessed March 23, 2021.
3. Chou ST, Fasano RM. Management of Patients with Sickle Cell Disease Using Transfusion Therapy: Guidelines and Complications. Hematology/oncology Clinics of North America. 2016 Jun;30(3):591-608. DOI: 10.1016/j.hoc.2016.01.011.