Dyno Therapeutics, Inc. announced the launch of its Dyno eCap 1 capsid product, an eye adeno-associated virus (AAV) gene delivery vector.
Let’s start with this company.
Founded in 2018 and based in Watertown, Massachusetts, Dyno Therapeutics is a techbio company focused on developing artificial intelligence (AI) applications to engineer AAV capsid designs that surpass current in vivo gene delivery vectors.
The goal: to expand the range of diseases (including in the eye) treatable with genetic medicines.
See here for a look at its other AAV capsid: the Dyno bCap 1, designed for the brain.
Exactly how is this being done?
Per the company, its novel CapsidMap platform is designed to “systemically and rapidly optimize AAV capsids” by focusing on improving four key areas:
- Tissue targeting ability
- Packaging size
- Immune evasion
- Manufacturability
The platform’s repeatable process includes:
- Designing capsid protein sequences to be synthesized on DNA printers and assembled into a capsid library for testing.
- Tracking DNA barcodes to identify individual capsid variants with properties for potential therapeutic use.
- Training machine learning models to predict capsid protein sequence functioning (via a fitness landscape)
- Searching the landscape for improved capsids to make current gene therapies more effective and enable new disease treatments
Now talk about this product.
Per Dyno, the Dyno eCap 1 capsid is essentially designed to provide significantly improved delivery (vs other externally engineered capsids) to the eye, including transduction throughout multiple layers of the retina.
Give me specifics on those layers.
The capsid has (per the company) demonstrated its ability to consistently transduce cell types across the following layers:
- Rod cells
- Bipolar cells
- Retinal ganglion cells (RGCs)
So what kind of ocular diseases would this impact?
Dyno co-founder and Chief Scientific Officer (CSO) Adrian Veres, MD, PhD, stated that such an effective delivery to the eye’s cells (and across the retina) is crucial for enabling gene therapies to treat:
- Wet age-related macular degeneration (AMD)
- Geographic atrophy (GA)
- Retinitis pigmentosa (RP)
To note, it is delivered via intravitreal injection.
And how was it tailored for ocular gene delivery?
Using its Capsid Map platform, the company conducted a large measurement of transduction and spatial location across the capsid sequence landscape.
This was done in order to map the various properties of the ocular capsid sequence space from which the Dyno eCap 1 vector could be identified.
And against a standard AAV2 capsid?
Compared to that standard capsid type, Dyno reported that its capsid has exhibited an 80-fold better transduction to the retina.
Gotcha. So what if I want to license this for my gene therapy program?
Click here and scroll down.