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Find Drugs for Genetic Disease in Preclinical Development

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            Lead Product(s): Inclacumab

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 December 06, 2020

            Details:

            In vitro study results demonstrated that inclacumab has the potential to be a best-in-class P-selectin inhibitor for reducing the frequency of VOCs in patients with SCD.

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            Lead Product(s): TSHA-103

            Therapeutic Area: Genetic Disease Product Name: TSHA-103

            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 December 03, 2020

            Details:

            TSHA-103 is an AAV-9-based gene therapy in development for SLC6A1-related epilepsy. TSHA-103 joins portfolio of rare pediatric disease and orphan drug designated product candidates including TSHA-101, TSHA-102 , TSHA-104 and TSHA-118.

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            Lead Product(s): ABX-002

            Therapeutic Area: Genetic Disease Product Name: ABX-002

            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 December 01, 2020

            Details:

            Autobahn has gained alignment with FDA on the initial Phase 1a/b clinical trial protocol for ABX-002 as a monotherapy treatment for a rare neurological disorder, adrenomyeloneuropathy (AMN).

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            Lead Product(s): PLX-300

            Therapeutic Area: Genetic Disease Product Name: PLX-300

            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 November 25, 2020

            Details:

            PLX-300 is a novel, small molecule found in many plants as a deaminated product of phenylalanine. If a new drug application (NDA) for PLX-300 is approved, the Company is eligible to receive a priority review voucher that may be sold or transferred to others.

            Sanford Burnham Prebys Medical Discovery Institute

            • Deals

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            Lead Product(s): Tissue non-specific alkaline phosphatase

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Soft Bones

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Funding November 19, 2020

            Details:

            Grant to support a study looking to determine the efficacy of viral vector delivery of mineral-targeted tissue non-specific alkaline phosphatase (TNAP) to treat late onset or adult HPP.

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            Lead Product(s): MMA-101,ImmTOR

            Therapeutic Area: Genetic Disease Product Name: MMA-101

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

            Partner/Sponsor/Collaborator: Selecta Biosciences

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 19, 2020

            Details:

            MMA-101 is an AAV-based gene therapy in development for the treatment of isolated methylmalonic acidemia due to methylmalonyl-CoA mutase gene mutations. AskBio and Selecta expect to start a Phase 1 trial of MMA-101 and ImmTOR for patients with MMA in the first half of 2021.

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            Lead Product(s): AVR-RD-06

            Therapeutic Area: Genetic Disease Product Name: AVR-RD-06

            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 November 17, 2020

            Details:

            Positive clinical data out as far as 3.5 years across a broad lysosomal disorder gene therapy pipeline were presented showcasing AVR-RD-02 for Gaucher disease type 1, AVR-RD-01 for Fabry disease, AVR-RD-04 for cystinosis.

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            Lead Product(s): Taurursodiol,Sodium Phenylbutyrate

            Therapeutic Area: Genetic Disease Product Name: AMX0035

            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 November 16, 2020

            Details:

            AMX0035 is designed to target the ER stress, and preclinical data suggest that it may be a promising approach to halt the irreversible progression of optic nerve atrophy in patients with Wolfram syndrome.