GBIO Shareholder/Stockholder Letter Transcript:
2023
Annual
Report
To our shareholders,
Last year was transformational for Generation Bio. We achieved
foundational breakthroughs leading us to focus on the development
of two distinct but complementary platforms: a cell-targeted lipid
nanoparticle (ctLNP) delivery system designed to selectively target
previously unreachable tissues and cell types while avoiding off-target
uptake, and a novel immune-quiet DNA (iqDNA) cargo that has been
shown to evade the innate immune response to DNA that has held
non-viral therapeutics back for decades. We are now actively
optimizing these platforms and using them to build our portfolio.
We believe the capabilities of our highly selective and potent ctLNP
delivery system could enable in vivo delivery of therapeutics to T cell
and to hematopoietic stem cell (HSC), transforming the current ex vivo
treatment paradigm, which is highly complex, lengthy, and expensive, to a much simpler, shorter, and
lower cost in vivo process. We are advancing a wholly owned program to address sickle cell disease
and beta-thalassemia using this approach for HSCs, and we continue to develop our work with our
partner Moderna to be able to name a first indication in T cells.
We believe that iqDNA is ideally suited to create a durable, redosable hemophilia A therapy.
Hemophilia A is the first of many disease areas where delivery of iqDNA to hepatocytes in the liver
could potentially apply. We are also keenly interested in exploring the profile of iqDNA in T cells and
HSCs, where we believe its unique immune-quiet profile may unlock a very large set of opportunities
that require either long-term DNA expression or the insertion of large DNA sequences.
To allow us to reach our critical development milestones for these platforms, we made the difficult
decision last year to reshape our company, yielding a smaller yet still strong and capable team of
approximately 120 scientists, engineers, and professionals. Through this process and further
reductions in operational expenditures, we have extended our cash runway into the second half of
2027, which affords us time to move forward in the portfolio development process.
I am so grateful to our current team, members of our organization who were impacted by our
reorganization, our board, and our investors for all we have achieved and all we have ahead of us.
We are well positioned and excited to reach critical development milestones for our ctLNP and iqDNA
platforms, as well as important proof points for portfolio programs in T cells, HSCs, and hepatocytes
this year.
Thank you for your continued support as we advance our mission to unlock the full potential of
genetic medicine.
Sincerely,
GEOFF MCDONOUGH
President and Chief Excutive Officer
4/24/2024 Letter Continued (Full PDF)