CSBio CSBio

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Find Drugs for Rare Diseases and Disorders in Preclinical Development in UNITED STATES

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            GC1126A is a novel ADAMTS13 mutein designed to evade autoantibodies with an extended half-life, which is investigated for the treatment of thrombotic thrombocytopenic purpura.

            Lead Product(s): GC1126A

            Therapeutic Area: Rare Diseases and Disorders Product Name: GC1126A

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 05, 2023

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            Under the agreement, Alexion expand its rare disease gene therapy portfolio by gaining Pfizer’s early-stage preclinical gene therapy programmes. The agreement furthers Alexion's commitment to advancing next-generation genomic medicines.

            Lead Product(s): Gene Therapy

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Alexion Pharmaceuticals

            Deal Size: $1,000.0 million Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement September 20, 2023

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            AB Institut de Neurociències

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            Details:

            Cannabidiol, a chemical compound from the cannabis plant, that help improvement in social and motor problems, a reduction in epilepsy and an increase in life expectancy in Leigh syndrome mouse models.

            Lead Product(s): Cannabidiol

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 14, 2023

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            The funding will be used for a novel Adeno-Associated Virus (AAV) gene therapy, GA-002, aimed at curing GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM).

            Lead Product(s): GA-002

            Therapeutic Area: Rare Diseases and Disorders Product Name: GA-002

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Solve GNE

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement August 14, 2023

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            Details:

            Under the agreement, Alexion will expand its rare disease gene therapy portfolio by gaining Pfizer’s early-stage preclinical gene therapy programmes. The agreement furthers Alexion's commitment to advancing next-generation genomic medicines.

            Lead Product(s): Gene Therapy

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Alexion Pharmaceuticals

            Deal Size: $1,000.0 million Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement July 28, 2023

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            Under the terms of the agreement, Sangamo has received a non-exclusive license to combine a Voyager TRACER capsid with Sangamo’s ZF-TRs (zinc finger transcriptional regulator) designed to treat prion disease.

            Lead Product(s): Undisclosed

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Sangamo Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement June 29, 2023

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            Two new gene therapy trials at UT Southwestern and Children’s Health funded through the consortium will test treatments for multiple sulfatase deficiency and Charcot-Marie-Tooth disease type 4J (CMT4J). Each clinical trial will include between eight and 12 patients.

            Lead Product(s): Undisclosed

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 27, 2023

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            Details:

            The agreement aims for the clinical development of NephroDI’s asset, NDI-5001, a novel kidney-targeted small molecule AMPK activator, as a potential therapy for X-linked congenital Nephrogenic Diabetes Insipidus (NDI).

            Lead Product(s): NDI-5001

            Therapeutic Area: Rare Diseases and Disorders Product Name: NDI-5001

            Highest Development Status: Preclinical Product Type: Small molecule

            Partner/Sponsor/Collaborator: McQuade Center for Strategic Research and Development

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement June 22, 2023

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            Details:

            The financing will fund completion of a first-in-man proof of concept study in subjects with Usher1B and further development of AAV intein program and entry into the clinic for Stargardt disease with AAVantgarde's two proprietary, AAV-based large gene delivery platforms.

            Lead Product(s): Usher1B-based Gene Therapy

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undislcosed

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Atlas Venture

            Deal Size: $65.2 million Upfront Cash: Undisclosed

            Deal Type: Series A Financing June 06, 2023

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            Details:

            Company will donate licenses to NAV® AAV8 and NAV® AAV9 from its NAV® Technology Platform to enable AMP® BGTC's development of these programs aimed at addressing ultra rare diseases i.e. Mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio syndrome.

            Lead Product(s): Undisclosed

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 15, 2023

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