Sickle Cell Disease

The Biden-Harris administration has announced an initiative to improve access to gene therapies for sickle cell disease under the Cell and Gene Therapy Access Model. This voluntary model, developed by the Center for Medicare and Medicaid Innovation, seeks to enhance access to these therapies,...

Imara Inc (NASDAQ: IMRA) shares are falling after interim analyses of its Ardent Phase 2b trial of tovinontrine (IMR-687) in sickle cell disease (SCD) and Forte Phase 2b trial in beta-thalassemia.  Data from the sickle cell disease study interim analysis demonstrated that...

The European Commission (EC) has approved Global Blood Therapeutics Inc's (NASDAQ: GBT) Oxbryta (voxelotor) for hemolytic anemia due to sickle cell disease (SCD) in adults and pediatric patients 12 years of age and older as monotherapy or in combination with hydroxycarbamide (...

The FDA has placed a partial clinical hold on Bluebird bio Inc's (NASDAQ: BLUE) lovotibeglogene autotemcel (lovo-cel) gene therapy program for sickle cell disease (SCD) for patients under the age of 18.  The partial, temporary suspension relates to an ongoing...

The FDA has granted accelerated approval to Global Blood Therapeutics Inc's (NASDAQ: GBT) supplemental application seeking approval for Oxbryta (voxelotor) tablets for sickle cell disease (SCD) in children ages 4 to less than 12 years. This approval expands the previously...

Global Blood Therapeutics Inc (NASDAQ: GBT) priced $300 million Convertible Senior Notes due 2028 in a private offering.  The offering size was increased from the previously announced offering size of $250 million.  Initial purchasers have an option to purchase up to an...

Bluebird bio Inc (NASDAQ: BLUE) announced updated results from its Phase 1/2 HGB-206 study of lovotibeglogene autotemcel (lovo-cel; formerly LentiGlobin for SCD, bb1111) gene therapy for sickle cell disease. The updated data included further analyses from its pivotal cohort, HGB-206...

Fulcrum Therapeutics Inc (NASDAQ: FULC) has announced results from the 20mg and 30mg dose cohorts in healthy adult volunteers in its Phase 1 trial of FTX-6058 for sickle cell disease and other hemoglobinopathies, such as beta-thalassemia. The Company also shared new preclinical...

Imara Inc (NASDAQ: IMRA) has announced a change to the primary endpoint for the Ardent Phase 2 trial of tovinontrine (IMR-687) in sickle cell disease. Imara is a biopharmaceutical company focused on therapeutics for rare inherited genetic disorders of hemoglobin. The change follows...

Jefferies initiated coverage of Global Blood Therapeutics Inc (NASDAQ: GBT) with a Buy rating and $68 price target. The analyst sees an upside of almost 145%. Oxbryta's implied sickle cell disease (SCD) market share is only 5%-6% at the stock's current valuation. ...

The FDA has accepted Global Blood Therapeutics Inc's (GBT) (NASDAQ: GBT) supplemental application seeking accelerated approval for Oxbryta (voxelotor) for sickle cell disease (SCD) in children ages 4 to 11 years. The agency has also accepted a marketing application for a new age...

Vertex Pharmaceuticals Inc (NASDAQ: VRTX) and Arbor Biotechnologies have announced a new collaboration to develop ex vivo engineered cell therapies. The new agreement between Arbor and Vertex builds upon the companies' first partnership established in 2018.  Related:...

Imara Inc (NASDAQ: IMRA) has priced its previously announced underwritten public offering of shares at $6.00 per share for gross proceeds of $50 million. The offering will close by July 16. Underwriters have an option to purchase up to $7.5 million of additional shares. Morgan Stanley,...

Forma Therapeutics Holdings Inc (NASDAQ: FMTX) announced new data from its ongoing Phase 1 trial of etavopivat (FT-4202) in sickle cell disease. Data were presented at the Annual European Hematology Association (EHA) 2021 Virtual Congress. The presentation included initial data from...

The FDA has lifted the clinical hold on Phase 1/2 HGB-206 and Phase 3 HGB-210 studies of Bluebird Bio Inc's (NASDAQ: BLUE) LentiGlobin for sickle cell disease and two Phase 3 studies of betibeglogene autotemcel gene therapy for transfusion-dependent β-thalassemia. The...