GBIO Shareholder/Stockholder Letter Transcript:
Annual
Report
2024
CHANGING WHAT S
POSSIBLE FOR
PEOPLE LIVING
WITH T CELL-DRIVEN
AUTOIMMUNE
DISEASE
To our shareholders,
I am pleased to share with you the signi cant progress Generation Bio has made over
the past year and our strategic vision for the future. We have made substantial strides
in developing our nucleic acid delivery platform and have reached a critical in ection
point in our path to delivering transformative treatments to patients.
In 2024, we demonstrated that our cell-targeted lipid nanoparticle (ctLNP) drives potent and selective T cell delivery,
allowing us to reach T cell targets without affecting other immune cell types. This selective delivery may allow us to
access targets that can powerfully modulate T cell activity and restore immune balance. Additionally, our ctLNP is
designed to be redosed and compatible with multiple nucleic acid cargos.
By pairing our targeted delivery system with small interfering RNA (siRNA) to achieve precise knockdown of intracellular
targets, we aim to create therapeutics that modulate T cell function without impacting the broader immune system.
Signi cant classes of drugs in immune and in ammation indications modulate T cells, but their impact is often
limited due to off-target effects. We believe that by combining the selective delivery of our ctLNP technology with
the genetic precision of siRNA, we can reach high-value targets that cause T cell-driven autoimmune diseases but
are undruggable by conventional methods. This approach has the potential to offer predictable pharmacology and
expand the therapeutic index, potentially creating more powerful T cell-speci c therapies with fewer side effects.
We are prioritizing high-value, undruggable targets for indications where we believe our approach could uniquely
improve upon the standard of care.
We are excited to move Generation Bio toward the clinic by deploying our ctLNP to deliver siRNA to T cells. We plan
to submit our rst investigational new drug (IND) application in the second half of 2026.
Looking ahead, we believe T cells are just the beginning. Our ctLNP is a modular delivery system designed to
selectively reach many other cell types and therapeutic areas. This versatility positions us to explore a wide range of
applications beyond T cell-driven diseases.
We have a strong organization with expertise in key functions, and we are bringing in selected new capabilities
critical to achieving our mission. Our team s continuity, dedication and experience are the foundation of our success,
and we are grateful for their contributions.
In conclusion, Generation Bio is at the forefront of creating rst-in-class targeted siRNA therapeutics to address T celldriven autoimmune disease. We believe our innovative ctLNP-siRNA technology, strategic target selection, and strong
intellectual property portfolio position us for continued success.
Thank you for your continued support and con dence in Generation Bio. Together, we are making meaningful
progress towards changing what s possible for people living with T cell-driven autoimmune disease.
Sincerely,
Geoff McDonough
President and Chief Executive Officer
4/23/2025 Letter Continued (Full PDF)