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            Development Status

            Details:

            The company intends to use the net proceeds to support the clinical development of BB-301, including the natural history lead-in study and the Phase 1b/2a BB-301 for the treatment of dysphagia associated with oculopharyngeal muscular dystrophy.

            Lead Product(s): BB-301

            Therapeutic Area: Rare Diseases and Disorders Product Name: BB-301

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

            Partner/Sponsor/Collaborator: JMP Securities

            Deal Size: $30.0 million Upfront Cash: Undisclosed

            Deal Type: Public Offering August 11, 2023

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            Details:

            The company intends to use the net proceeds to support the clinical development of BB-301, including the natural history lead-in study and the Phase 1b/2a BB-301 for the treatment of dysphagia associated with oculopharyngeal muscular dystrophy.

            Lead Product(s): BB-301

            Therapeutic Area: Rare Diseases and Disorders Product Name: BB-301

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

            Partner/Sponsor/Collaborator: JMP Securities

            Deal Size: $30.0 million Upfront Cash: Undisclosed

            Deal Type: Public Offering August 08, 2023

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            Details:

            BB-301 is a novel, modified AAV9 capsid expressing bifunctional construct promoting co-expression of both codon-optimized Poly-A Binding Protein Nuclear-1 and two small inhibitory RNAs against mutant PABPN1 is currently in development for Oculopharyngeal Muscular Dystrophy.

            Lead Product(s): BB-301

            Therapeutic Area: Rare Diseases and Disorders Product Name: BB-301

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 26, 2023

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            BB-301 is currently in development for Oculopharyngeal Muscular Dystrophy. BB-301 results in robust inhibition of mutant PABPN1 and concomitant replacement of the codon-optimized PABPN1 protein. The treatment restores muscle strength and muscle weight.

            Lead Product(s): BB-301

            Therapeutic Area: Rare Diseases and Disorders Product Name: BB-301

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 23, 2023

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            Details:

            The Company intends to use the net proceeds from this financing for the clinical development of BB-301, including the natural history lead-in study and the Phase 1b/2a BB301 treatment study.

            Lead Product(s): BB-301

            Therapeutic Area: Rare Diseases and Disorders Product Name: BB-301

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: JMP Securities

            Deal Size: $18.0 million Upfront Cash: Undisclosed

            Deal Type: Public Offering September 16, 2022

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            Details:

            The company intends to use the proceeds for development of BB-301, including the natural history lead-in study and Phase 1b/2a BB-301 treatment study, for continued advancement of development activities, for general corporate purposes and for strategic growth opportunities.

            Lead Product(s): BB-301

            Therapeutic Area: Rare Diseases and Disorders Product Name: BB-301

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: JMP Securities

            Deal Size: $18.0 million Upfront Cash: Undisclosed

            Deal Type: Public Offering September 12, 2022

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            Details:

            The BB-301 Tissue Transduction Study is the first of three studies to be conducted in large animals, with the overall goal of providing data to submit an investigational new drug application to the U.S. Food and Drug Administration, a mandatory step to conduct clinical trials.

            Lead Product(s): BB-301

            Therapeutic Area: Rare Diseases and Disorders Product Name: BB-301

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 23, 2020

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            Details:

            BB-301 is an internally optimized, AAV-based gene therapy agent that can both silence the expression of mutated, disease-causing genes (to slow, or halt, the underlying mechanism of disease progression) and replace the mutant genes with normal, "wild type" genes.

            Lead Product(s): BB-301

            Therapeutic Area: Rare Diseases and Disorders Product Name: BB-301

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 08, 2020

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