RAG1

Mustang Bio Announces First Patient Successfully Treated by Ex Vivo Lentiviral Gene Therapy to Treat RAG1 Severe Combined Immunodeficiency

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Wednesday, July 27, 2022
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LV-RAG1 is exclusively licensed by Mustang for the development of MB-110, a first-in-class ex vivo lentiviral gene therapy for the treatment of RAG1-SCID.

Key Points: 
  • LV-RAG1 is exclusively licensed by Mustang for the development of MB-110, a first-in-class ex vivo lentiviral gene therapy for the treatment of RAG1-SCID.
  • The license agreement grants Mustang rights to certain additional lentiviral gene therapies being developed in Dr. Staals lab.
  • Severe combined immunodeficiency (SCID) due to complete RAG1 deficiency is a rare, genetic severe combined immunodeficiency disorder caused by null mutations in the RAG1 gene resulting in less than 1% of wild type V(D)J recombination activity.
  • Mustang has partnered with top medical institutions to advance the development of CAR T therapies across multiple cancers, as well as lentiviral gene therapies for severe combined immunodeficiency.

Mustang Bio Reports Third Quarter 2021 Financial Results and Recent Corporate Highlights

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Friday, November 12, 2021
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WORCESTER, Mass., Nov. 12, 2021 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (Mustang) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced financial results and recent corporate highlights for the third quarter ended September 30, 2021.

Key Points: 
  • WORCESTER, Mass., Nov. 12, 2021 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (Mustang) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced financial results and recent corporate highlights for the third quarter ended September 30, 2021.
  • In November 2021, Mustang announced that the company was awarded a grant of approximately $2 million from NCI of the National Institutes of Health.
  • Research and development expenses including license acquisitions were $14.7 million for the third quarter of 2021, compared to $8.3 million for the third quarter of 2020.
  • General and administrative expenses were $2.4 million for the third quarter of 2021, compared to $2.2 million for the third quarter of 2020.

Mustang Bio Announces Exclusive Worldwide License Agreement with Leiden University Medical Centre for Clinical-Stage Lentiviral Gene Therapy with Curative Potential for RAG1 Severe Combined Immunodeficiency

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Wednesday, November 10, 2021
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WORCESTER, Mass., Nov. 10, 2021 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (“Mustang”) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced that the company has executed an exclusive license agreement with Leiden University Medical Centre (“LUMC”) for a first-in-class ex vivo lentiviral gene therapy for the treatment of RAG1 severe combined immunodeficiency (“RAG1-SCID”).

Key Points: 
  • Dr. Staal will continue the development of additional lentiviral gene therapies in his lab, to which Mustang Bio has rights under the agreement.
  • The RAG1-SCID therapy expands the pipeline of ex vivo lentiviral gene therapies currently in development at Mustang.
  • The Companys lead programs, MB-107 and MB-207, are being investigated for the treatment of X-linked severe combined immunodeficiency (XSCID).
  • Mustang is establishing itself as the leader in developing treatments for patients with severe combined immunodeficiency, an area of high unmet need.

Cellectis Presents Initial Preclinical Data on Two Novel Gene Therapies for Patients with RAG1 Severe Combined Immunodeficiency (SCID) and Hyper IgE syndrome at ESGCT 2021

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Tuesday, October 19, 2021
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ProfessorCathomensteam at University of Freiburgwill bepresentingencouraging pre-clinical data that supports further evaluation ofCellectis .HEAL platform,aninnovative gene therapyplatformthat usesa genome editing approachbased onTALEN ,fortwoproduct candidatestargetingprimary immunodeficiencies: RAG1forSevere Combined Immunodeficiency (SCID) andSTAT3 forHyperIgEsyndrome.

Key Points: 
  • ProfessorCathomensteam at University of Freiburgwill bepresentingencouraging pre-clinical data that supports further evaluation ofCellectis .HEAL platform,aninnovative gene therapyplatformthat usesa genome editing approachbased onTALEN ,fortwoproduct candidatestargetingprimary immunodeficiencies: RAG1forSevere Combined Immunodeficiency (SCID) andSTAT3 forHyperIgEsyndrome.
  • Last May,duringCellectis Innovation Days ,the Companyrevealed its new .HEAL platform, a novel hematopoietic stem cell gene therapy that aims to address debilitating genetic diseases.
  • Newborns with RAG1 SCID have extremely low levels of B and T cells and a severe risk of recurrent, life-threatening infections.
  • The precise replacement of the mutated gene enabled the corrected RAG1 gene to be expressed at its natural timing and stage of cell development.