-- Collaboration to leverage Orna's
differentiated lipid nanoparticle (LNP) delivery solutions for
patients with SCD and TDT --
-- Orna to receive an upfront payment and is
eligible to receive potential pre-clinical, clinical, and
commercialization milestone payments and royalties --
WATERTOWN, Mass., Jan. 7, 2025
/PRNewswire/ -- Orna Therapeutics (through its wholly owned
subsidiary ReNAgade Therapeutics Inc.) announced a three-year
strategic research collaboration with Vertex Pharmaceuticals
Incorporated (Nasdaq: VRTX) to utilize Orna's novel and proprietary
LNP delivery solutions to enhance Vertex's efforts in developing
next generation gene editing therapies for patients with SCD and
TDT.
"Vertex is a leader in delivering next-generation approaches to
treating hemoglobinopathies, and we are excited to collaborate with
them to develop in vivo therapies that leverage our
proprietary technologies to achieve unprecedented delivery to
HSCs," commented Amit Munshi, Chief
Executive Officer of Orna. "Today's collaboration further validates
our industry leading extra-hepatic LNP delivery chemistries and
highlights the importance of delivery to enable the next wave of
RNA medicines."
Ansbert Gadicke, M.D., Chairman of Orna and Managing Partner of
MPM BioImpact added, "Today's collaboration leveraging Orna's
industry-leading non-viral HSC delivery represents our second major
partnership and is testament to the breadth and potential of the
Company's platform. We look forward to partnering with Vertex to
develop transformative therapies for SCD and TDT, while
simultaneously advancing our lead panCAR™ pipeline programs in
autoimmune and oncology. This deal further solidifies Orna's
leadership in next generation RNA medicines and has the potential
to deliver large-scale impact to patients."
About the Collaboration
Under the terms of the
agreement, Orna will receive upfront payments of $65 million, including an investment in the form
of a convertible note, and is eligible to receive up to
$635 million based upon the
achievement of specified pre-clinical, research, development,
regulatory and commercial milestones related to SCD/TDT products.
Additionally, Orna is further eligible to receive up to
$365 million in additional option
fees and milestones per product for up to ten additional products
if Vertex options rights in additional indications. Orna will be
eligible to receive tiered royalties on future net sales of any
products that may result from this collaboration. Vertex is funding
the three-year research collaboration and holds an option to extend
the research collaboration term.
About Orna Therapeutics
Orna Therapeutics is dedicated
to designing and delivering a new class of fully engineered
circular RNA (oRNA®) therapeutics to unlock the
potential of RNA medicine to treat diseases anywhere in the body.
Orna's circular RNA transcripts have advantages over traditional
mRNA approaches, including simplified production, improved
formulation into lipid nanoparticles, and superior protein
expression. Its industry-leading LNP-based delivery systems and
comprehensive editing programs position Orna to advance novel RNA
medicines with vast potential to transform patient care. To learn
more, visit www.ornatx.com and follow Orna Therapeutics on X and
LinkedIn.
Orna Therapeutics Investor Contact:
Alex Lobo
Precision AQ
alex.lobo@precisionaq.com
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SOURCE Orna Therapeutics
