HSC

BioLineRx Announces Acceptance of Two Poster Presentations on APHEXDA® (motixafortide) for CD34+ Hematopoietic Stem Cell (HSC) Mobilization in Patients with Multiple Myeloma at the 2024 Tandem Meetings of ASTCT® and CIBMTR®

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Friday, February 16, 2024

Phase 1 study results demonstrated an extended PD effect with complete receptor occupancy by motixafortide starting at a concentration of 3nM.

Key Points: 
  • Phase 1 study results demonstrated an extended PD effect with complete receptor occupancy by motixafortide starting at a concentration of 3nM.
  • In the GENESIS trial, post-hoc subgroup analyses based on baseline characteristics and risk factors for impaired HSC mobilization demonstrated a consistent benefit of motixafortide + G-CSF over placebo + G-CSF mobilization for all patients.
  • Poster Presentations at the 2024 Tandem Meetings: Transplantation & Cellular Therapy Meetings of the ASTCT and the CIBMTR.
  • Title: Motixafortide Enables Consistent, Robust Hematopoietic Stem Cell Collection (HSC) across Populations with Increased Impaired HSC Mobilization: A Sub-Group Analysis of the Genesis Study
    Presenter: Zachary D. Crees, MD, Washington University School of Medicine in St. Louis

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)

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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.

Orchard Therapeutics Announces First Patient Randomized in Registrational Trial of OTL-203 for MPS-I Hurler Syndrome

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Monday, February 5, 2024

TOKYO, LONDON and BOSTON, Feb. 05, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced the first patient has been randomized at the M Health Fairview Masonic Children’s Hospital in a registrational trial evaluating the efficacy and safety of OTL-203, an investigational hematopoietic stem cell (HSC) gene therapy, in patients with the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). The trial, referred to as HURCULES, compares treatment with OTL-203 to standard of care with allogeneic hematopoietic stem cell transplant (HSCT), and is expected to enroll 40 MPS-IH patients at sites across the U.S. and Europe.

Key Points: 
  • Approximately 60 percent of children born with MPS-I have the most severe subtype, MPS-IH, also called Hurler syndrome, and rarely live past the age of 10 when untreated.
  • Current treatment options for MPS-IH include allogeneic hematopoietic stem cell transplant (HSCT) and enzyme replacement therapy (ERT), both of which have significant limitations.
  • One patient experienced an acute hypersensitivity reaction that was considered probably related to HSC gene therapy.
  • The viral vector integration profile was comparable with other Orchard Therapeutics lentiviral-based HSC gene therapy studies, and all participants had a stable and highly polyclonal repertoire.

Humboldt Seed Company Debuts First High-Potency Triploid Cannabis Seeds

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Tuesday, January 30, 2024

SAN FRANCISCO, Jan. 30, 2024 /PRNewswire/ -- Humboldt Seed Company (HSC), the largest licensed cannabis seed seller in California, today announced the release of its OG Kush Triploid and Donutz Triploid cannabis seeds to the U.S. market.

Key Points: 
  • High-THC triploid seed release expands Humboldt Seed Company's catalog of novel, terpene-rich strains.
  • SAN FRANCISCO, Jan. 30, 2024 /PRNewswire/ -- Humboldt Seed Company (HSC), the largest licensed cannabis seed seller in California, today announced the release of its OG Kush Triploid and Donutz Triploid cannabis seeds to the U.S. market.
  • "This seed release has been a long time coming," said Benjamin Lind, co-founder and chief science officer of Humboldt Seed Company.
  • "I had a chance to compare the triploid and diploid plants at the Humboldt Seed Company 2023 phenotype hunt.

Orchard Therapeutics Announces Agreement with the Beneluxa Consortium Enabling Reimbursed Access to Libmeldy

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Thursday, January 25, 2024

BOSTON and LONDON, Jan. 25, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a global gene therapy leader, today announced the company has reached an agreement with the Beneluxa Initiative on Pharmaceutical Policy (Beneluxa) enabling reimbursed access to Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, approved for the treatment of early-onset metachromatic leukodystrophy (MLD).

Key Points: 
  • BOSTON and LONDON, Jan. 25, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a global gene therapy leader, today announced the company has reached an agreement with the Beneluxa Initiative on Pharmaceutical Policy (Beneluxa) enabling reimbursed access to Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, approved for the treatment of early-onset metachromatic leukodystrophy (MLD).
  • With more than a cumulative 250 patient-years of follow-up, Libmeldy was generally well-tolerated, with no treatment-related serious adverse events or deaths.
  • “We are pleased to have reached an agreement with the Beneluxa consortium that enables sustainable access to treatment.
  • Multiple eligible MLD patients are currently in the treatment process and expected to receive Libmeldy in the coming months.

Kyowa Kirin successfully completes acquisition of Orchard Therapeutics, a global gene therapy leader for rare diseases

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Wednesday, January 24, 2024

TOKYO and LONDON and BOSTON, Jan. 24, 2024 (GLOBE NEWSWIRE) -- Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151) a Japan-based global specialty pharmaceutical company (J-GSP) discovering and developing novel medicines utilizing the latest biotechnology, and Orchard Therapeutics plc (Orchard Therapeutics, Nasdaq: ORTX), a global gene therapy leader, today announced Kyowa Kirin has successfully completed the acquisition of Orchard Therapeutics.

Key Points: 
  • TOKYO and LONDON and BOSTON, Jan. 24, 2024 (GLOBE NEWSWIRE) -- Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151) a Japan-based global specialty pharmaceutical company (J-GSP) discovering and developing novel medicines utilizing the latest biotechnology, and Orchard Therapeutics plc (Orchard Therapeutics, Nasdaq: ORTX), a global gene therapy leader, today announced Kyowa Kirin has successfully completed the acquisition of Orchard Therapeutics.
  • The acquisition of all outstanding shares of Orchard Therapeutics by way of a Scheme of Arrangement procedure under the UK Companies Act 2006 was completed on January 24, 2024, and Orchard Therapeutics has become a wholly-owned subsidiary of Kyowa Kirin.
  • “We are truly excited about the acquisition of Orchard Therapeutics, a leading provider of HSC gene therapy.
  • Kyowa Kirin anticipates significant synergies with Orchard Therapeutics and plans to announce its 2024 Financial Guidance in conjunction with its 2023 earnings on 7 February.

Kyowa Kirin Successfully Completes Acquisition of Orchard Therapeutics, a Global Gene Therapy Leader for Rare Diseases

Retrieved on: 
Wednesday, January 24, 2024

Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151) a Japan-based global specialty pharmaceutical company (J-GSP) discovering and developing novel medicines utilizing the latest biotechnology, and Orchard Therapeutics plc (Orchard Therapeutics, Nasdaq: ORTX), a global gene therapy leader, today announced Kyowa Kirin has successfully completed the acquisition of Orchard Therapeutics.

Key Points: 
  • Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151) a Japan-based global specialty pharmaceutical company (J-GSP) discovering and developing novel medicines utilizing the latest biotechnology, and Orchard Therapeutics plc (Orchard Therapeutics, Nasdaq: ORTX), a global gene therapy leader, today announced Kyowa Kirin has successfully completed the acquisition of Orchard Therapeutics.
  • The acquisition of all outstanding shares of Orchard Therapeutics by way of a Scheme of Arrangement procedure under the UK Companies Act 2006 was completed on January 24, 2024, and Orchard Therapeutics has become a wholly-owned subsidiary of Kyowa Kirin.
  • “We are truly excited about the acquisition of Orchard Therapeutics, a leading provider of HSC gene therapy.
  • Kyowa Kirin anticipates significant synergies with Orchard Therapeutics and plans to announce its 2024 Financial Guidance in conjunction with its 2023 earnings on 7 February.

ElevateBio to Highlight Growth of its Genetic Medicine Business Driven By Gene Editing and Manufacturing Partnerships at the 42nd Annual J.P. Morgan Healthcare Conference

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Monday, January 8, 2024

Chairman and Chief Executive Officer, David Hallal, will provide a business update at the 42nd Annual J.P. Morgan Healthcare Conference on January 8, 2024 at 9:30 am PT.

Key Points: 
  • Chairman and Chief Executive Officer, David Hallal, will provide a business update at the 42nd Annual J.P. Morgan Healthcare Conference on January 8, 2024 at 9:30 am PT.
  • Announced two transformative R&D collaborations with Moderna and Novo Nordisk to discover and develop gene editing therapeutics.
  • BaseCamp is ElevateBio’s genetic medicine process development and cGMP business enabling biopharmaceutical partners with its end-to-end capabilities.
  • Developed additional C-base editors for gene knock-outs in novel cell therapies and A-base editors for in vivo gene editing therapeutics.

Molecular Partners Provides Updates at 42nd Annual J.P. Morgan Healthcare Conference

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Sunday, January 7, 2024

53 LR Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, today announced it will present a business overview and provide its 2024 outlook at the 42nd Annual J.P. Morgan Healthcare Conference.

Key Points: 
  • 53 LR Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, today announced it will present a business overview and provide its 2024 outlook at the 42nd Annual J.P. Morgan Healthcare Conference.
  • Data were presented at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition in December 2023.
  • Molecular Partners continues to progress its RDT platform and portfolio of projects, both in-house and in partnership with Novartis.
  • In addition to these updates, Novartis has returned the rights to the ensovibep program, previously under investigation for the treatment of COVID-19, to Molecular Partners.