SCD

AMO Pharma Announces Collaboration with Population Health Research Institute to Advance Proof of Concept Clinical Trial to Assess Efficacy of Tideglusib in Treatment of Arrhythmogenic Cardiomyopathy

Retrieved on: 
Thursday, February 15, 2024

Enrollment is set to begin in mid-2024 at 20 sites across Canada and will include a total of 120 participants who will be randomized in a 1:1 ratio.

Key Points: 
  • Enrollment is set to begin in mid-2024 at 20 sites across Canada and will include a total of 120 participants who will be randomized in a 1:1 ratio.
  • The TaRGET trial is a critical first step in evaluating this potential."
  • This scarring increases the risk of dangerously fast heart rhythms that can lead to sudden cardiac death (SCD).
  • An implantable cardioverter defibrillator (ICD) is recommended for prevention of SCD for ACM patients considered at high risk for SCD.

AMO Pharma Announces Collaboration with Population Health Research Institute to Advance Proof of Concept Clinical Trial to Assess Efficacy of Tideglusib in Treatment of Arrhythmogenic Cardiomyopathy

Retrieved on: 
Thursday, February 15, 2024

Enrollment is set to begin in mid-2024 at 20 sites across Canada and will include a total of 120 participants who will be randomized in a 1:1 ratio.

Key Points: 
  • Enrollment is set to begin in mid-2024 at 20 sites across Canada and will include a total of 120 participants who will be randomized in a 1:1 ratio.
  • The TaRGET trial is a critical first step in evaluating this potential."
  • This scarring increases the risk of dangerously fast heart rhythms that can lead to sudden cardiac death (SCD).
  • An implantable cardioverter defibrillator (ICD) is recommended for prevention of SCD for ACM patients considered at high risk for SCD.

European Commission Approves First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), for the Treatment of Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT)

Retrieved on: 
Tuesday, February 13, 2024

ZUG, Switzerland and BOSTON, Feb. 13, 2024 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, announced today that the European Commission has granted conditional marketing authorization to CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene edited therapy. CASGEVY is approved for the treatment of patients who are 12 years of age and older with severe sickle cell disease (SCD) characterized by recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT), for whom hematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen matched related HSC donor is not available.

Key Points: 
  • “The approval by the European Commission is yet another important regulatory milestone underscoring the potentially transformative benefit of CASGEVY for patients with severe sickle cell disease and transfusion-dependent beta thalassemia,” said Samarth Kulkarni, Chairman and Chief Executive Officer of CRISPR Therapeutics.
  • Vertex is working closely with national health authorities in the European Union (EU) to secure access for eligible patients as quickly as possible.
  • Through this work, they have secured early access for eligible TDT patients in France ahead of the national reimbursement process.
  • There are currently three activated ATCs in the EU with plans to activate a total of approximately 25 centers across Europe.

European Commission Approves First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

Retrieved on: 
Tuesday, February 13, 2024

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Commission has granted conditional marketing authorization to CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the European Commission has granted conditional marketing authorization to CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy.
  • CASGEVY is the only genetic therapy approved for SCD and TDT patients in the European Union (EU) and with this approval, there are now more than 8,000 patients potentially eligible for treatment.
  • Through this work, Vertex has secured early access for eligible TDT patients in France ahead of the national reimbursement process.
  • Vertex continues to engage with hospitals experienced in stem cell transplantation to establish a network of independently operated authorized treatment centers (ATCs) for the administration of CASGEVY.

SeaStar Medical Granted Canadian Patent with Broad Claims Covering the Selective Cytopheretic Device Technology

Retrieved on: 
Thursday, February 1, 2024

DENVER, Feb. 01, 2024 (GLOBE NEWSWIRE) -- SeaStar Medical Holding Corporation (Nasdaq: ICU), a medical device company developing proprietary solutions to reduce the consequences of hyperinflammation on vital organs, announces the issuance by the Canadian Intellectual Property Office of Canadian Patent No.

Key Points: 
  • DENVER, Feb. 01, 2024 (GLOBE NEWSWIRE) -- SeaStar Medical Holding Corporation (Nasdaq: ICU), a medical device company developing proprietary solutions to reduce the consequences of hyperinflammation on vital organs, announces the issuance by the Canadian Intellectual Property Office of Canadian Patent No.
  • This new patent has broad claims covering the Company’s Selective Cytopheretic Device (SCD) technology.
  • Its issuance expands SeaStar Medical’s international patent rights and complements existing U.S. patent rights.
  • This newly issued Canadian patent, titled Cytopheretic Cartridge and Use Thereof, provides coverage of SCD cartridges for treating activated leukocytes and activated platelets, as well as their use in processing activated leukocytes and platelets.

Emmaus Life Sciences Receives Marketing Authorization for Puerto Rico

Retrieved on: 
Friday, February 2, 2024

TORRANCE, Calif., Feb. 2, 2024 /PRNewswire/ -- Emmaus Life Sciences, Inc. (OTCQX: EMMA), a leader in sickle cell disease (SCD) treatment, today announced that it has received marketing authorization from the Puerto Rico Department of Health for Endari® (L-glutamine oral powder).

Key Points: 
  • TORRANCE, Calif., Feb. 2, 2024 /PRNewswire/ -- Emmaus Life Sciences, Inc. (OTCQX: EMMA), a leader in sickle cell disease (SCD) treatment, today announced that it has received marketing authorization from the Puerto Rico Department of Health for Endari® (L-glutamine oral powder).
  • This approval marks a significant milestone in Emmaus' mission to improve the lives of people with SCD around the world and provides access to this important therapy for the patients living with SCD in Puerto Rico.
  • "We are thrilled to bring Endari® to Puerto Rico, where the SCD community has faced significant challenges in accessing innovative treatments," said George Sekulich, Co-President of Emmaus Life Sciences.
  • Emmaus is committed to working with healthcare providers and payers in Puerto Rico to ensure that Endari® is accessible to all eligible patients.

Solvias to Perform Release Testing on World's First CRISPR-based Gene-Editing Therapy

Retrieved on: 
Tuesday, January 23, 2024

KAISERAUGST, Switzerland, Jan. 23, 2024 /PRNewswire/ -- Solvias, a global provider of chemistry, manufacturing, and control (CMC) analytics, announced today that it will perform analytical release testing services on the world's first CRISPR/Cas9 genome-edited cell therapy. The company has signed a long-term agreement with Vertex Pharmaceuticals for CASGEVY™ (exagamglogene autotemcel or exa-cel) which received U.S. Food & Drug Administration (FDA) approval for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises.

Key Points: 
  • KAISERAUGST, Switzerland, Jan. 23, 2024 /PRNewswire/ -- Solvias, a global provider of chemistry, manufacturing, and control (CMC) analytics, announced today that it will perform analytical release testing services on the world's first CRISPR/Cas9 genome-edited cell therapy.
  • The companies' collaboration included establishing the testing methods that will be scaled for commercializing CASGEVY.
  • Solvias also has invested significantly in preparing one of its global facilities to support the commercial release work for this transformative therapy.
  • "Solvias is honored to be playing a critical role in delivering this breakthrough therapy to patients.

Solvias to Perform Release Testing on World's First CRISPR-based Gene-Editing Therapy

Retrieved on: 
Tuesday, January 23, 2024

KAISERAUGST, Switzerland, Jan. 23, 2024 /PRNewswire/ -- Solvias, a global provider of chemistry, manufacturing, and control (CMC) analytics, announced today that it will perform analytical release testing services on the world's first CRISPR/Cas9 genome-edited cell therapy. The company has signed a long-term agreement with Vertex Pharmaceuticals for CASGEVY™ (exagamglogene autotemcel or exa-cel) which received U.S. Food & Drug Administration (FDA) approval for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises.

Key Points: 
  • KAISERAUGST, Switzerland, Jan. 23, 2024 /PRNewswire/ -- Solvias, a global provider of chemistry, manufacturing, and control (CMC) analytics, announced today that it will perform analytical release testing services on the world's first CRISPR/Cas9 genome-edited cell therapy.
  • The companies' collaboration included establishing the testing methods that will be scaled for commercializing CASGEVY.
  • Solvias also has invested significantly in preparing one of its global facilities to support the commercial release work for this transformative therapy.
  • "Solvias is honored to be playing a critical role in delivering this breakthrough therapy to patients.

CRISPR Therapeutics Announces U.S. Food and Drug Administration (FDA) Approval of CASGEVY™ (exagamglogene autotemcel) for the Treatment of Transfusion-Dependent Beta Thalassemia

Retrieved on: 
Tuesday, January 16, 2024

ZUG, Switzerland and BOSTON, Jan. 16, 2024 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older.

Key Points: 
  • “We are pleased with the approval of CASGEVY in TDT well ahead of the PDUFA date,” said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics.
  • “The approval is a reflection of the power and versatility of the CRISPR platform to bring a potentially curative treatment option to patients suffering from this devastating disease.”
    The administration of CASGEVY requires experience in stem cell transplantation; therefore, our partner Vertex Pharmaceuticals Incorporated is engaging with experienced hospitals to establish a network of independently operated, authorized treatment centers (ATCs) throughout the U.S. to offer CASGEVY to patients.
  • All nine ATCs activated in the U.S. are able to offer CASGEVY to eligible patients with TDT and sickle cell disease (SCD).
  • Additional ATCs will be activated in the coming weeks and a complete list of ATCs can be accessed at CASGEVY.com.

Vertex Announces US FDA Approval of CASGEVY™ (exagamglogene autotemcel) for the Treatment of Transfusion-Dependent Beta Thalassemia

Retrieved on: 
Tuesday, January 16, 2024

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the U.S. Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that the U.S. Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older.
  • “On the heels of the historic FDA approval of CASGEVY for sickle cell disease, it is exciting to now secure approval for TDT well ahead of the PDUFA date,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex.
  • All nine ATCs activated in the U.S. are able to offer CASGEVY to eligible patients with TDT and sickle cell disease (SCD).
  • Additional ATCs will be activated in the coming weeks and a complete list of ATCs can be accessed at CASGEVY.com .