Scd

bluebird bio to Present Data from Gene and Cell Therapy Programs During the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition

Retrieved on: 
Thursday, November 5, 2020
Infectious diseases, General Health, Health, Other Health, Branches of biology, Health sciences, Life sciences, Health in Africa, Hematopathology, Sickle cell disease, Biotechnology, Bluebird bio, Gene therapy, Stearoyl-CoA desaturase-1, Thalassemia, Scd

(Nasdaq: BLUE) announced today that data from its gene and cell therapy programs for sickle cell disease (SCD), transfusion-dependent beta-thalassemia (TDT) and multiple myeloma (MM) will be presented, including seven oral presentations, at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, taking place virtually from December 5-8, 2020.

Key Points: 
  • (Nasdaq: BLUE) announced today that data from its gene and cell therapy programs for sickle cell disease (SCD), transfusion-dependent beta-thalassemia (TDT) and multiple myeloma (MM) will be presented, including seven oral presentations, at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, taking place virtually from December 5-8, 2020.
  • Updated results from patients in Group C of the companys Phase 1/2 HGB-206 study of LentiGlobin for SCD gene therapy (bb1111) will be presented.
  • LentiGlobin for SCD (bb1111) is an investigational gene therapy being studied as a potential treatment for SCD.
  • bluebird bios clinical development program for LentiGlobin for SCD includes the ongoing Phase 1/2 HGB-206 study and the ongoing Phase 3 HGB-210 study.

Cyclerion Announces Phase 2 STRONG-SCD Study Results in Patients with Sickle Cell Disease

Retrieved on: 
Wednesday, October 14, 2020
Health in Africa, Branches of biology, Hematopathology, RTT, Sickle cell disease, Clinical medicine, Medical specialties, Stearoyl-CoA desaturase-1, Scd, University of Pittsburgh Epidemiology Data Center

Results did not demonstrate adequate activity to support further internal clinical development.

Key Points: 
  • Results did not demonstrate adequate activity to support further internal clinical development.
  • Cyclerion intends to complete its analysis of the study results and present or publish them in a future forum.
  • We would like to thank all of the sickle cell patients who participated in the STRONG-SCD study, the SCD advocacy community, and the investigators and study staff who focused their time and efforts on helping us better understand the potential of olinciguat in sickle cell disease, saidPeter Hecht, Ph.D., Chief Executive Officer of Cyclerion.
  • For more information about Cyclerion, please visit https://www.cyclerion.com/ and follow us on Twitter ( @Cyclerion ) and LinkedIn ( www.linkedin.com/company/cyclerion ).

GBT Announces New Employment Inducement Grants

Retrieved on: 
Monday, October 5, 2020
Branches of biology, Selectins, Proteins, Medical specialties, Health in Africa, Hematopathology, RTT, Sickle cell disease, Scd, Stearoyl-CoA desaturase-1, P-selectin, E-selectin

These awards were made under GBTs Amended and Restated 2017 Inducement Equity Plan (the Plan).

Key Points: 
  • These awards were made under GBTs Amended and Restated 2017 Inducement Equity Plan (the Plan).
  • The above-described awards were each granted as an inducement material to the employees entering into employment with the company in accordance with NASDAQ Listing Rule 5635(c)(4), and were granted pursuant to the terms of the Plan.
  • 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.
  • GBT is also advancing its pipeline program in SCD with inclacumab, a p-selectin inhibitor in development to address pain crises associated with the disease.

bluebird bio’s LentiGlobin™ for Sickle Cell Disease Gene Therapy (bb1111) Granted Priority Medicines (PRIME) Designation by European Medicines Agency

Retrieved on: 
Wednesday, September 23, 2020
Research, Other Health, General Health, Pharmaceutical, Oncology, Hospitals, Genetics, Clinical trials, Science, Biotechnology, Stem cells, Health, Other Science, Branches of biology, Articles, Health, RTT, Health in Africa, Biotechnology, Gene delivery, Rare diseases, Sickle cell disease, Gene therapy, Orphan drug, Scd

Even with recent progress to deliver new medicines for sickle cell disease, there remains a profound unmet need for people living with SCD.

Key Points: 
  • Even with recent progress to deliver new medicines for sickle cell disease, there remains a profound unmet need for people living with SCD.
  • LentiGlobin for SCD received orphan medicinal product designation from the European Commission for the treatment of SCD.
  • The U.S. FDA granted orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation and rare pediatric disease designation for LentiGlobin for SCD.
  • Were putting our care and expertise to work across a spectrum of disorders, including cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using three gene therapy technologies: gene addition; cell therapy and (megaTAL-enabled) gene editing.

GBT and Sickle Cell Disease Association of America to Host 9th Annual Sickle Cell Disease (SCD) Therapeutics Conference

Retrieved on: 
Wednesday, September 9, 2020
Health in Africa, Hematopathology, RTT, Sickle cell disease, Branches of biology, Scd, Clinical medicine, Sickle Cell Disease Association of America, Stearoyl-CoA desaturase-1, Medical specialties

SOUTH SAN FRANCISCO, Calif. and BALTIMORE, Sept. 09, 2020 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) and theSickle Cell Disease Association of America, Inc.(SCDAA) will host the 9thAnnualSickle Cell Disease (SCD) Therapeutics ConferenceonTuesday, September 15.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif. and BALTIMORE, Sept. 09, 2020 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) and theSickle Cell Disease Association of America, Inc.(SCDAA) will host the 9thAnnualSickle Cell Disease (SCD) Therapeutics ConferenceonTuesday, September 15.
  • 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.
  • We have a responsibility to improve health outcomes for the SCD community, said Ted W. Love, M.D., president and CEO of GBT.
  • 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.

GBT Announces New Employment Inducement Grants

Retrieved on: 
Friday, September 4, 2020
Proteins, Branches of biology, Selectins, Health in Africa, Hematopathology, RTT, Sickle cell disease, Scd, Stearoyl-CoA desaturase-1, P-selectin, E-selectin

These awards were made under GBTs Amended and Restated 2017 Inducement Equity Plan (the Plan).

Key Points: 
  • These awards were made under GBTs Amended and Restated 2017 Inducement Equity Plan (the Plan).
  • The above-described awards were each granted as an inducement material to the employees entering into employment with the company in accordance with NASDAQ Listing Rule 5635(c)(4), and were granted pursuant to the terms of the Plan.
  • 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.
  • GBT is also advancing its pipeline program in SCD with inclacumab, a p-selectin inhibitor in development to address pain crises associated with the disease.

GBT Announces Participation in Upcoming Investor Conferences

Retrieved on: 
Thursday, September 3, 2020
Proteins, Branches of biology, Selectins, Health in Africa, Hematopathology, RTT, Sickle cell disease, Scd, Stearoyl-CoA desaturase-1, P-selectin, E-selectin

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Key Points: 
  • ET;
    Morgan Stanley 18th Annual Global Healthcare Conference on September 16 at 10:15 a.m.
  • 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.
  • GBT is also advancing its pipeline program in SCD with inclacumab, a p-selectin inhibitor in development to address pain crises associated with the disease.
  • In addition, GBTs drug discovery teams are working on new targets to develop the next generation of treatments for SCD.

Study: Medical Marijuana Associated with Fewer Hospitalizations for Individuals Living with Sickle Cell Disease

Retrieved on: 
Thursday, August 13, 2020
Health in Africa, Hematopathology, RTT, Sickle cell disease, Branches of biology, Clinical medicine, Medical specialties, Scd

WASHINGTON, Aug. 13, 2020 /PRNewswire/ --Individuals with sickle cell disease (SCD) who receive medical marijuana to treat pain may require fewer visits to the hospital, according to a new study in Blood Advances.

Key Points: 
  • WASHINGTON, Aug. 13, 2020 /PRNewswire/ --Individuals with sickle cell disease (SCD) who receive medical marijuana to treat pain may require fewer visits to the hospital, according to a new study in Blood Advances.
  • When this occurs, individuals living with SCD experience severe pain events which may drive them to seek emergency care.
  • Dr. Curtis and her team examined data from 50 individuals with SCD at the Adult Sickle Cell Program at Yale New Haven Hospital who were certified for medical marijuana use.
  • Medical marijuana is associated with significant stigma, and stigma is already a big part of the life of a person with sickle cell," said Dr. Curtis.

New Recommendations Aim to Increase Capacity for Specialized Care for Adults Living with Sickle Cell Disease

Retrieved on: 
Wednesday, August 12, 2020
Health in Africa, Hematopathology, RTT, Sickle cell disease, Branches of biology, Clinical medicine, Scd, Medical specialties, Draft:Sickle Cell Disease in the Caribbean

Since the universal adoption of newborn screening in the United States in 2006, care for children living with SCD has improved.

Key Points: 
  • Since the universal adoption of newborn screening in the United States in 2006, care for children living with SCD has improved.
  • Other barriers to high-quality care for adults living with SCD include challenges relating to health insurance cost and coverage, socioeconomic status, and co-existing conditions.
  • "I have taken care of numerous people who never even knew there was a sickle cell specialist.
  • The ASH Sickle Cell Disease Initiative includes development of SCD clinical practice guidelines , expansion of education and training efforts, focused federal policy efforts, and establishment of the Sickle Cell Disease Coalition .

GBT Announces Participation in Upcoming Investor Conferences

Retrieved on: 
Thursday, July 30, 2020
Proteins, Branches of biology, Life sciences, Selectins, Health in Africa, Hematopathology, RTT, Sickle cell disease, Transmembrane receptors, Scd, P-selectin, Genuity

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Key Points: 
  • ET; and
    Canaccord Genuity 40th Annual Growth Virtual Conference on August 13 at 1:30 p.m.
  • 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.
  • GBT is also advancing its pipeline program in SCD with inclacumab, a p-selectin inhibitor in development to address pain crises associated with the disease.