Gene therapy biotech looking to bypass viral delivery wins investor approval, bagging Series A
The viral vectors that carried the first generation of gene therapies into the clinic have proven to be a troublesome lot for a variety of reasons. And now one of the biotechs brewing new ways to circumvent those obstacles in gene therapy 2.0 has a major new funding round to fuel the work.
Code Biotherapeutics announced Tuesday that it raised $75 million via a Series A round, courtesy of a group of blue-chip investors. CEO Brian McVeigh — a GSK veteran who used to run the pharma’s BD unit — told Endpoints News that the funds will last the biotech well into 2024. And so far, there are some big plans for that funding, including pipeline and in-house manufacturing expansion, plus progressing the company’s two disease programs further in preclinical into IND-enabling studies.
The biotech said in a statement that Northpond Ventures led the financing round, alongside Amgen Ventures, Hatteras Venture Partners and UCB Ventures. Previous investors who jumped in on the round included NEA, 4BIO Capital and Takeda Ventures.
The biotech has been focusing on non-viral gene therapy delivery since the biotech officially started in 2020, launching out of stealth with a $10 million seed round last year in a current focus on type 1 diabetes and Duchenne muscular dystrophy, or DMD. As McVeigh explained, the main technology behind Code Bio is a drug delivery scaffold made of synthetic DNA strands, and has 18 ends of base pairs 3 prime and 5 prime. Combined with a targeting moiety, the scaffold can be brought to a specific cell type — and bring along a genetic medicine payload to the cell, too.
As McVeigh further elaborated, going the synthetic scaffold approach would allow for one key differentiator — re-dosing. Current AAV vectors are only good for one dose, as patient’s immune systems either build up an immunity to the virus or are already naturally immune.
So far, Code Bio has been working on data to show that its targeting system works — that the scaffold can go into muscle cells for DMD and alpha/beta cells in the liver for type 1 diabetes. The next step — at least for the delivery system — is to test the efficacy in in vivo models for both diseases.
Additionally, Code is moving forward with an approach that will allow for the scaffold to take an entire copy of the dystrophin gene — the one that is dysregulated in patients with DMD and the largest gene — into the body. Beyond that, McVeigh said the biotech is also looking at a smaller version called Becker’s gene (about two-thirds the length of the dystrophin gene) and its own microdystrophin.
While it’s too soon to say which of Code’s options will move forward, McVeigh added that it has seen a waning effect in AAV-based microdystrophin approaches. However, if only Code Bio’s microdystrophin approach makes it through to the finish line and the FDA’s thumbs-up, it would still be able to re-dose patients — and then it may not matter what DMD approach comes through clinicals and regulatory review.
The biotech is one of several players to try and get movement in the non-viral gene therapy delivery space, and most of them have substantial backing of some sort. Generation Bio closed a nine-figure IPO last June in a bid to push preclinical work for a non-viral gene therapy in liver and retina diseases. Michael Ehlers — the former R&D chief at Biogen and ex-CSO, neuroscience at Pfizer — is part of another effort as an advisor at Xalud Therapeutics.
This new Series A is also just a few months after Code Bio scored a partnership with Takeda — worth up to $2 billion — back in February for four unspecified rare disease projects. And according to McVeigh, while it is still very early in the relationship, it has been great so far. The biotech also has two more pharma partnerships — but as to who those pharmas are, that remains a mystery.