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Find Clinical Drug Pipelines for Rare Diseases and Disorders

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            Lead Product(s): Engenious vectors,HSC gene therapy

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: 5AM Ventures

            Deal Size: $70.0 million Upfront Cash: Undisclosed

            Deal Type: Series A Financing February 11, 2021

            Details:

            Company’s Engenious™ vectors Delivered without Stem Cell Collection or Myeloablative Conditioning in Outpatient and Low-Resource Settings, Enabling Access Worldwide.

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            Lead Product(s): Elamipretide

            Therapeutic Area: Rare Diseases and Disorders Product Name: MTP-131

            Highest Development Status: Phase II/ Phase III Product Type: Peptide

            Partner/Sponsor/Collaborator: H.C. Wainwright & Co

            Deal Size: $4.7 million Upfront Cash: Undisclosed

            Deal Type: Public Offering February 10, 2021

            Details:

            The Company intends to use the net proceeds from this offering to fund the continued clinical development of elamipretide and for working capital and general corporate purposes.

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            Lead Product(s): Daratumumab,Hyaluronidase-fihj,Bortezomib

            Therapeutic Area: Rare Diseases and Disorders Product Name: Darzalex Faspro

            Highest Development Status: Approved Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 15, 2021

            Details:

            Accelerated approval of DARZALEX FASPRO-based combination regimen supported by the Phase 3 ANDROMEDA study demonstrating a significantly higher hematologic complete response rate in this rare and serious blood cell disorder.

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            Lead Product(s): Elamipretide

            Therapeutic Area: Rare Diseases and Disorders Product Name: MTP-131

            Highest Development Status: Phase II/ Phase III Product Type: Peptide

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 06, 2021

            Details:

            DCN suggested that, based on the short half-life of elamipretide, a short-term withdrawal period could be sufficient to measure durable effect on functional or structural cardiac endpoints by demonstrating that improvement is not dependent on continuous exposure to the drug.

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            Lead Product(s): Undisclosed

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Discovery Platform Product Type: Undisclosed

            Partner/Sponsor/Collaborator: GlaxoSmithKline

            Deal Size: $1,150.0 million Upfront Cash: Undisclosed

            Deal Type: Collaboration December 18, 2020

            Details:

            The collaboration will combine Adrestia’s world leading, synthetic viability platform with GSK’s deep scientific expertise in human genetics, functional genomics, screening, and bioinformatics.

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            Lead Product(s): INO-3107

            Therapeutic Area: Rare Diseases and Disorders Product Name: INO-3107

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 23, 2020

            Details:

            The open-label, multicenter INO-3107 Phase 1/2 trial will evaluate the efficacy, safety, tolerability, and immunogenicity of INO-3107 in subjects who have required at least two surgical interventions per year for the past three years for the removal of associated papilloma.

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            Lead Product(s): Tesofensine,Metoprolol Tartrate

            Therapeutic Area: Rare Diseases and Disorders Product Name: Tesomet

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 23, 2020

            Details:

            Tesomet was well-tolerated in hypothalamic obesity patients throughout the duration of the 48-week trial, with no clinically meaningful differences in heart rate or blood pressure observed. All patients who entered the OLE study completed it.

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            Lead Product(s): Exosome-based therapeutics

            Therapeutic Area: Rare Diseases and Disorders Product Name: Undisclosed

            Highest Development Status: Discovery Product Type: Undisclosed

            Partner/Sponsor/Collaborator: Evox Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Collaboration November 17, 2020

            Details:

            The collaboration will run over three years and will capitalise on the world-class exosome research environment in Oxford, leveraging exosome therapeutics expertise and resources from both Evox and the University to drive innovation within rare disease drug development.