Sustainable Differentiation & Competitive Advantages
Our potential to become the most impactful company in the gene therapy industry is driven by three unique value drivers at 4D:
- Robust, industry-leading vector discovery platform: Our Therapeutic Vector Evolution platform is able to generate highly optimized, customized and proprietary AAV vectors for any tissue in the body. This will allow us to develop better products, and to create products to target many diseases that cannot be touched with the current first-generation native AAV vectors. Our founding scientist, Dr. David Schaffer, has pioneered this platform at UC Berkeley, and together with the team at 4D we are taking the discovery capabilities to a whole new level. Over the next decade, we expect 4D vectors to become the gold standard for the industry.
- Extensive development expertise with viral vector gene therapeutics: Our team has deep industry-leading experience translating viral vectors into the clinic, and developing them in well-designed and executed clinical trials. Dr. Kirn has led clinical R&D programs with 10 different viral vector products, involving six different vector platforms, over 30 clinical trials, and over 800 patients with life-threatening diseases. This clinical-regulatory experience will accelerate the development of our 4D products.
- Diversified, sustainable and value-generating business model: The value of our business is driven by our product pipeline. 4D products will be developed in three ways. First, we will develop a number of products all the way to the market ourselves. Second, we plan to enter into co-development and profit-share partnerships with complementary companies. Third, we are selectively licensing our customized vectors for delivery of specific transgene payloads for the treatment of specific diseases. To date we have licensing partnerships with the following companies:
Competition: 4D Sustainable Competitive Advantages
4D AAV vectors have important advantages that differentiate them from currently available vectors:
- Customization for specific target tissues in patients: Naturally occurring vectors such as AAV2, AAV8 and AAV9 were discovered as lab contaminants or monkey infections. In contrast, 4D vectors are discovered through directed evolution for delivery to a specific tissue by the optimal clinical route of administration.
- Selected by Directed Evolution from a massive variant pool: We select our vectors from a pool of roughly 100 million novel 4D variants that were created over 15 years by 20 different methods. In contrast, our competitors use vectors that were selected from a handful of naturally occurring vectors. 4D vectors are therefore selected from a proprietary vector pool that is 1 million times more diverse.
- Optimization for delivery through multiple complimentary mechanisms: Because Directed Vector Evolution allows nature to select the best vector capsids for delivery to the target tissue, the approach is not biased towards any single step in the process from administration to clinical efficacy. Our vectors therefore can be optimized at multiple complimentary steps.
- Antibody evasion: 4D vectors can be selected for evasion of pre-existing antibodies in the general population. This should translate into better delivery, better efficacy and more patients amenable to treatment. In contrast, standard available vectors can often be neutralized in a majority of patients in the population, and more than 50% of patients are excluded from many trials due to pre-existing antibodies.
- Strong composition-of-matter patentability: 4D vectors are selected from a proprietary pool of roughly 100 million novel variants. Composition-of-matter patent claims should therefore be enabled. In contrast, the patentability of naturally occurring serotypes has been called into question by a recent Supreme Court ruling in the US.
- Leadership in Directed Evolution and Therapeutic Vector Evolution:
- Directed Vector Evolution was pioneered by our co-founder Professor David Schaffer at UC Berkeley over the last 15 years. Over $15 Million dollars of grant funding supported the development of this vector discovery platform.
- At 4DMT, we’ve built on this original platform to create Therapeutic Vector Evolution. In addition to Dr. Schaffer’s expertise as CSO, 4D has hired experienced personnel from Dr. Schaffer’s lab. Finally, the 4D AAV variant pool consists of roughly 100 million novel variants. To our knowledge, this is the largest and most diverse variant pool in the world. Our vectors have been constructed by over 20 different methodologies in order to maximize diversity. We therefore have the expertise, the personnel and the reagents to maximize the potential of this discovery platform.