Monthly Archives: September 2021

GBT and Sickle Cell Disease Association of America to Host 10th Annual Sickle Cell Disease (SCD) Therapeutics Conference

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 10th 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 10th 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 10th 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.

# # #
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
 
   

MARAC Issues Updated COVID-19 Guidance


August 26, 2021 – The Sickle Cell Disease Association of America Medical and Research Advisory Committee has released two statements with updated COVID-19 guidance.
MARAC recommendations are making a minor shift to emphasize a more individualized approach:

  • Continue to recommend vaccination against COVID. For booster dose of vaccine, currently SCD is not eligible but expect changes in CDC guidance.
  • Continue to recommend general precautions: wearing masks, keeping physical distancing, good ventilation, and washing hands. These are public health measures.
  • Recommend neutralizing monoclonal antibodies (mAb) – (Regeneron) as early treatment for mild symptomatic COVID in individuals with SCD [<10d after the test, age 12y+, wt 88 lb+ (40kg+), not hospitalized, not newly on oxygen]. Encourage getting tested if you have COVID symptoms. Possible prophylactic treatment with Regeneron antibodies if exposed to COVID.
  • No general recommendation for all individuals with SCD to stay home nor all to return to in-person activities. Help patients and families make individualized assessments of risk and trade-offs of returning to work or school in-person. Factors to consider were listed in MARAC July 2020 “checklist for return to school” (plus vaccination status): the community’s rates of COVID and variants, vaccination status, family socioeconomic situation, ventilation and other protective measures in the building, mental health needs, educational needs.

Read the full statement for more information on the above.
Additionally, MARAC recommends that people with sickle cell disease should have access to monoclonal antibody (mAbs) treatment according to established guidelines, to prevent mild COVID from progressing to severe disease. Read the full recommendations for providers, policy makers and patients.
 

HCPLive® Announces Partnership With Sickle Cell Disease Association of America (SCDAA)

HCPLive® Announces Partnership With Sickle Cell Disease Association of America (SCDAA)
SCDAA’s mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to raise awareness and maximize patient quality of life.
(CRANBURY, N.J. – September 7, 2021) – HCPLive®, a multimedia platform dedicated to providing physicians with up-to-date specialty and disease-specific information to help them offer the best patient care, is thrilled to announce the addition of the Sickle Cell Disease Association of America (SCDAA) to its Strategic Alliance Partnership (SAP) program.
“The addition of the Sickle Cell Disease Association of America to our SAP program will allow us to further collaborate on initiatives that will positively impact the industry,” said Mike Hennessy Jr., president and CEO of MJH Life Sciences™, parent company of HCPLive®. “With a mission of advocating to raise awareness and maximize patient quality of life, SCDAA’s goals align directly with ours as a publication.”
As a leader in promoting and advancing initiatives focused on people affected by sickle cell conditions worldwide, SCDAA and its affiliate organizations pursue national health care objectives while demonstrating how community-based organizations can work. After more than 45 years, SCDAA continues to recognize how critical it is to raise awareness and gain support for both the social and public health aspects of sickle cell disease.
SCDAA’s national efforts focus on six broad areas:

  • Research
  • Public Health Education
  • Professional Health Education
  • Patient Services
  • Community Services
  • Support to Global Organizations and Practitioners

The SAP program builds a community of advocacy groups, medical associations, and medical institutions to foster collaboration and an open exchange of information among trusted peers, for the ultimate benefit of patients and their families. As part of this joint effort, HCPLive® will work with the partners to share exclusive information and improve patient outcomes. 
For the full list of HCPLive® SAP partners, click here. 
About HCPLive®  
HCPLive® is a comprehensive clinical news and information portal that provides physicians and other health care professionals with up-to-date specialty- and disease-specific resources to help them deliver better care to patients. Readers have access to breaking news, video interviews with physician experts, in-depth conference coverage, finance and practice management updates, insights and analysis from physician contributors and other resources. HCPLive® is a brand of MJH Life Sciences™, the largest privately held, independent, full-service medical media company in North America, dedicated to delivering trusted health care news across multiple channels.  
HCPLive®Media Contact  
Alyssa Scarpaci, 609-716-7777 
ascarpaci@mjhlifesciences.com