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From 'Rare' to 'Everywhere': Generation Bio's Genetic Revolution

Generation Bio's pioneering platform is revolutionizing the landscape of healthcare, offering hope to a broader range of patients, it is where innovative genetic medicine is reshaping the future of healthcare.

  • Generation Bio's pioneering platform is revolutionizing the landscape of healthcare, offering hope to a broader range of patients, it is where innovative genetic medicine is reshaping the future of healthcare.
  • The company is on a mission to make durable, adaptable treatments accessible to individuals living with rare and prevalent diseases. 
  • It was founded in 2016 and Geoff Mcdonough is the Chief Executive Officer of Generation Bio.

Do you know about gene therapy? Are you wondering how they 'gene'-rate hope? Curious about the brains behind the 'genius' in genetic therapy? If so, you're not alone in your curiosity.

All about Generation Bio

It is a visionary company co-founded by Mark Angelino and Robert Kotin. They are at the forefront of innovation, harnessing the potential of genetic medicine to provide durable, re- dosable treatments for individuals living with rare and prevalent diseases.

The mission at Generation Bio is to unlock the full promise of genetic medicine, not just for some diseases and some patients, but for all.  The manufacturing processes used currently for complex genetic therapies, like AAV-based gene therapies, simply are not able to produce enough doses to treat highly prevalent diseases.

This is one reason why most genetic medicines in development are for rare diseases.  Solving these manufacturing challenges could make access to genetic medicines widespread and sustainable.

Their pioneering platform has the potential to extend the power of gene therapy to many more diseases, affecting many more people, than what is possible with existing gene therapy technology. They are advancing a broad and expansive portfolio in both rare and prevalent diseases.

Imagine Generation Bio's approach to genetic medicine like building a sophisticated toolkit for healthcare; Three breakthroughs in construction, delivery, and manufacturing empower their mission to create a new class of scalable, re- dosable, non-viral genetic medicine for hundreds of millions of patients living with rare and prevalent diseases.

How does it work?

The company’s non-viral platform incorporates a novel DNA construct called closed-ended DNA, or ceDNA; a unique cell-targeted lipid nanoparticle delivery system, or ctLNP; and a highly scalable capsid-free manufacturing process that uses its proprietary cell-free rapid enzymatic synthesis, or RES, to produce ceDNA. 

In simpler terms, think of their DNA construct (ceDNA) as the blueprints, their delivery system (ctLNP) as the delivery truck, and their manufacturing process (RES) as the high-speed factory.  

The platform is designed to enable multi-year durability from a single dose, to deliver large genetic payloads, including multiple genes, to specific tissues, and to allow titration and redosing to adjust or extend expression levels in each patient.

Their aim to create a new class of genetic medicine that leaves no one behind calls for a manufacturing process that is efficient, reliable, and scalable. RES can be rapidly scaled to produce not millions, but hundreds of millions of doses to enable their vision: a future where genetic medicine is available to more people, living with more diseases, in more places around the world.


Generation Bio's remarkable journey in rewriting the future of healthcare is not only driven by innovation but also backed by significant investment. The company is funded by 12 investors, Moderna and Foresite Capital being the most recent investors, and has raised a total of $536.4M in funding over 5 rounds.

Their latest funding was raised from a Post-IPO Equity round and they have a post-money valuation in the range of $500M to $1B as of Jan 10, 2020, according to PrivCo. 

This financial foundation reflects the belief in their platform. In a nutshell, Generation Bio is rewriting the future of healthcare with innovative genetic medicine that's durable, adaptable, and accessible to all.

With their groundbreaking platform, they are bringing hope to a wider range of diseases and patients, and revolutionizing how we approach genetic therapies. When genetic disease touches a life, it affects a whole family. 

Generation Bio looks forward to the day when parents, grandparents, brothers, sisters, and spouses can spend less time focused on their loved one’s health and more time enjoying their loved one’s life.

Edited by Shruti Thapa