SEC Filings

10-K
AUDENTES THERAPEUTICS, INC. filed this Form 10-K on 03/13/2017
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Table of Contents

initiated cGMP manufacturing of our products in our facility in the second half of 2016. We have made and will continue to make significant investments to further optimize our manufacturing capabilities to cost-effectively produce high-quality AAV vectors at both clinical and commercial scale. We believe that our manufacturing processes, methods, expertise and facilities will give us a comprehensive manufacturing platform for production of our AAV product candidates at commercial scale.

We have a focused, passionate team with collective expertise in gene therapy, rare disease drug development and commercialization, and biologics manufacturing. Matthew Patterson, our President, Chief Executive Officer and Co-Founder, is a biotechnology leader with over 20 years of experience at Genzyme Corporation, BioMarin Pharmaceutical, Amicus Therapeutics and our company.

Our Strategy

Our strategy is to leverage the expertise of our team and the transformative potential of gene therapy technology to develop treatments that improve outcomes for patients with serious, life-threatening rare diseases. Key elements of our strategy are:

 

Focus on serving patients. We take pride in our efforts to harness the transformative potential of gene therapy to improve the lives of patients suffering from devastating rare diseases. We intend to continue to engage with patient advocacy groups to better understand the burden of disease and align our efforts with the needs of patients and caregivers.

 

Advance our lead product candidates through clinical development. We have an active IND for AT342 for the treatment of Crigler-Najjar, and we submitted an IND for AT132 for the treatment of XLMTM in the first quarter of 2017. Our collaborating institution, the University of Florida, has submitted an investigator sponsored IND to conduct a proof-of-concept study of AT982 delivered via intra-muscular injection, and we are conducting IND-enabling preclinical work for the systemic administration of AT982 for the treatment of Pompe disease and exploratory preclinical work evaluating intrathecal delivery of AT982.  We expect to have preliminary data from the AT132, AT342 and AT982 programs in the second half of 2017, and plan to file an IND for AT307 in the second half of 2017.

 

Continue to expand our pipeline with additional gene therapy product candidates targeting serious, life-threatening rare diseases. We intend to continue leveraging our expertise and focused selection criteria to expand our pipeline of product candidates. Our relationships with leading academic institutions and other rare disease companies are an important component of our strategy for sourcing additional product candidates.

 

Continue to build our proprietary manufacturing capabilities and invest in a state-of-the-art cGMP facility. We believe the quality, reliability and scalability of our gene therapy manufacturing approach will be a core competitive advantage crucial to our long-term success. We initiated cGMP manufacturing of our products in our facility in the second half of 2016.

Our Strengths

We believe our leadership position is based on our following strengths:

 

Rare disease expertise. Led by a management team with over 100 years of combined experience in rare diseases, we are building a fully integrated and industry-leading biotechnology company. Leveraging recent developments in gene therapy, we aim to provide durable and meaningful treatment options to patients suffering from rare monogenic diseases.

 

Highly focused selection criteria for development programs. We employ a disciplined approach to select and expand our pipeline of product candidates. We believe the application of our selection criteria enables the efficient, cost-effective and successful development of our product candidates.

 

Promising product candidate pipeline. On the basis of rigorous preclinical investigation, we are preparing to advance our product candidates into the clinic: AT132 for the treatment of XLMTM, AT342 for the treatment of Crigler-Najjar, AT982 for the treatment of Pompe disease and AT307 for the treatment of CASQ2-CPVT.

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