The 4D Approach to Product Development & Partnerships.
We believe in success through collaboration. We seek as partners world-leading teams that bring complementary capabilities and resources, with the goal of introducing potentially life-saving therapies to market more rapidly. Through collaborations, we can extend our reach beyond our internal pipeline to additional disease indications. Our partners augment our capabilities through their disease area expertise and networks; strong intellectual property on promising transgene payloads and/or regulatory elements; financial resources applied to product R&D and biologics commercialization expertise.
Our approach to partnering is open and flexible. We prefer a creative approach to deal structuring that optimizes the benefit for both parties. Together with our partner, we design and execute a development and commercialization plan for the business relationship and disease targets. Based on the therapeutic area and specific product(s) involved, we are open to either 1) a co-development relationship, or 2) and out-licensing arrangement.
4DMT has entered into multiple alliances, including with Pfizer, Roche, AGTC, uniQure and the Choroideremia Research Foundation. We also have grant funding from the Cystic Fibrosis Foundation.
We partner with great companies that have some or all of the following strengths:
- Disease area expertise and networks
- Strong intellectual property on promising transgene payloads and/or regulatory elements
- Financial resources to contribute to product R&D
- Biologics commercialization expertise
Our product development and commercialization model is flexible, and we adapt it to fit each partnership. We engage with our partners to design and execute the best R&D and commercialization plan for the specific business relationship and disease targets. We select from two general collaboration models:
- Co-development and profit-sharing relationship
- Out-licensing to partner in return for upfront, milestones and royalty payments
Our 2016 partnership with Pfizer is focused on the discovery and development of targeted and proprietary AAV vectors for use in gene therapy products for specific CARDIAC indications. Pfizer made an equity investment in 4DMT and holds a seat on the Board of Directors. 4DMT retains the rights to develop products for all other non-partnered transgenes and indications in the heart.
Our 2016 partnership with Roche is focused on the discovery and development of targeted and proprietary AAV vectors for use in gene therapy products for specific rare RETINAL disease indications. 4DMT retains the rights to develop products for all other non-partnered indications in the eye.
Our broad 2014 partnership with uniQure is focused on the discovery and development of targeted and proprietary AAV vectors for use in gene therapy products for diseases within the CENTRAL NERVOUS SYSTEM and LIVER. 4DMT retains the rights to develop products for indications that uniQure elects not to pursue.
Our 2015 partnership with Applied Genetic Technologies Corporation (AGTC) is to accelerate development of novel AAV-based gene therapies to treat rare RETINAL diseases. AGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop products initially focused on ophthalmology.
Our 2014 partnership agreement with Benitec Biopharma, an Australian biotechnology company with a proprietary platform that combines gene silencing and gene therapy technology, focuses on vectors for delivery of proprietary Benitec technology to treat RETINAL disease indications.
Our 2016 partnership with the Chorolderemia Research Foundation is to develop a gene therapy product optimized for intravitreal administration to treat Choroideremia, a hereditary retinal degenerative disease. The Foundation brings clinical expertise, a network of doctors, trial investigators and patients together to inform product clinical and trial design and execution.
Our 2015 grant from the Cystic Fibrosis Foundation is to support development of a gene therapy product optimized for aerosolized delivery to treat cystic fibrosis, a hereditary lung disease. The Foundation brings research and clinical expertise, a network of doctors, trial investigators and patients together to inform product design, R&D, clinical trial design and execution.