Biotechs Transforming Gene Therapy on DNA Day



Meet the Disruptors: Biotechs Transforming Gene Therapy on DNA Day


A Tribute to the Pioneers
DNA Day is an annual celebration to commemorate James Watson, Francis Crick, and  Rosalind Franklin’s discovery (1953) of the double-stranded structure responsible for heredity in humans and almost all other organisms. This ultimately enabled the Human Genome Project, which was completed in April of 2003, coincidentally marking the 50th anniversary of Watson, Crick, and Franklin’s discovery and the inception of DNA Day.

Both of these discoveries were necessary drivers in the evolution of gene therapies and personalized medicine and have enabled concepts of biotechnology wherein health disorders are treated or prevented by introducing healthy genes into a patient’s cells. While we’ve barely scraped the surface of gene therapies and the benefits they’ll bring to the table, research & development efforts are accelerating.

New gene therapy organizations are being propped up at an astounding pace. For DNA Day 2023, we’re highlighting 3 emerging biotech organizations looking to bring their novel gene therapies to in-human trials.


Aera Therapeutics (MA, US)

Aera Therapeutics came onto the scene with a splash, as they launched in February with the help of $193 million raised in combined Series A & B financings. Aera’s proprietary protein nanoparticle (PNP) delivery platform is built to package and transfer nucleic acids for therapeutic benefits. Their technology also includes a therapeutic enzyme platform built off of novel gene editing enzymes.

The safe and efficient delivery of genetic medicines is a common hurdle the biotech industry faces when it comes to development. Hence why we’re seeing so many platform companies emerge, solving the issues of drug delivery before embarking on the discovery of novel therapeutic assets. In this case, their PNP platform enables cargo delivery to various tissues, leaving few limitations when it comes to addressable therapeutic areas. Out-licensing of platform technologies is not uncommon, but with significant amounts of available capital, Aera is poised to begin developing its own novel therapeutic assets.


SpliceBio (Spain)

This European startup spun out from Princeton University is leveraging protein splicing technology to develop novel gene therapies and was founded in Barcelona. The oversubscribed €50 million series A financing round in February of 2022 was the largest ever for a Spanish biotech. SpliceBio is utilizing those funds to advance their lead program for the treatment of Stargardt disease toward clinical trials. Additionally, they’re working on expanding their pipeline to address other untreatable genetic disorders.
Their protein splicing platform was built to overcome issues in delivering large genes using AAV vectors. While ophthalmology is the main therapeutic area of focus for this emerging biotech, their platform has been validated for use in several other organs in addition to the retina. Having just recently been awarded the 2023 Biosuccess Award, keep a close eye on SpliceBio for key pipeline updates in the year ahead.


Dyno Therapeutics (MA, US)

Lo and behold, another biotech startup coming out of Cambridge! Although this one has taken a unique approach in its strategy. Dyno is another example of a platform company within the gene therapy space, supported by a $100 million Series A dating back to May 2021. Its CapsidMap platform leverages AI to support the design of novel AAV gene therapy vectors.

Why are they unique? Because they have elected not to keep the platform to themselves. Thus far, they have signed agreements with the likes of Roche, Astellas, Serapta Therapeutics, and Novartis to utilize the platform for the development of their own therapies. While Dyno has scientists in place for the development of their own pipeline, their ongoing collaborations occupy the majority of their current efforts. Thus far, they have not shied away from going against the grain and have demonstrated that AI can and will play an essential role in the future of life sciences.


Teamwork Makes the Dream Work

Unlike the small molecule space where we’ve amassed blueprints for drug development, the majority of gene therapy-related developments are happening for the first time. This means that collaboration will be key in accelerating the progress in what’s proving to be a very promising sector in health sciences.

Established pharma & biotech companies leaning on the technology of startups like Dyno Therapeutics are encouraging steps that will hopefully inspire future partnerships that will aid in bringing novel therapies to patients in need.


Authored by: Alec de la Durantaye, Product Marketing Manager, Zymewire

April 25, 2023 | Health & Science Days