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Origenis GmbH Announces Global License and Collaboration Agreement with Expansion Therapeutics for Novel Therapies Targeting RNA-mediated Diseases

Thursday, January 24th 2019 at 1:00pm UTC

– Companies Initiate Lead Optimization Program Leveraging the Origenis
AI Discovery Platform to Identify RNA-targeted Small Molecule Compounds
for Development –

MARTINSRIED, Germany–(BUSINESS WIRE)– Origenis announced today it has entered into a global license and
collaboration agreement with Expansion Therapeutics to research and
discover small molecule compounds targeting RNA-mediated diseases. As
part of the agreement, the companies will initiate a lead optimization
program which leverages the Origenis artificial intelligence (AI)
discovery platform to identify drug development candidates for
Expansion’s portfolio of RNA-targeted small molecule medicines.

“We are excited to apply our technology platform as well as our
interdisciplinary approach and chemical expertise to a novel class of
compounds supporting Expansion in their efforts to develop medicines for
RNA-mediated diseases,” Michael Almstetter, CEO of Origenis. “We have
already validated our platform’s target independence with results from
an initial feasibility phase using RNA-based targets. The continued
collaboration with Expansion provides an opportunity to further extend
our expertise into a unique therapeutic space that differentiates us
from other AI companies.”

The collaboration combines Origenis’ capabilities in compound design,
chemical synthesis, and screening with Expansion’s field-leading
expertise in the discovery and development of small molecules
interacting with ribonucleic acid (RNA). RNA is becoming more widely
recognized for playing a diverse role in cell biology and is implicated
in many diseases, making it an attractive therapeutic target.

Through the use of its proprietary AI drug discovery platform, MolMind®
and the MOREsystem®, in conjunction with other innovative embedded
technologies, Origenis will design, synthesize and characterize novel
molecules acting against different RNA-based targets. Origenis will
deliver identified development candidates with defined properties for
these targets to Expansion and will ensure the novelty by guiding the
process with Cippix®, a unique tool creating value from hidden chemical
and biological information embedded in patents. Throughout its drug
discovery process, Origenis employs interactive AI feedback learning to
ensure compounds have unique chemical properties and have been tested
for biological activity, toxicity and stability for real-world

Under the terms of the agreement, Expansion will receive rights to
develop products against defined molecular targets. Origenis will
receive research payments and is eligible for certain development

About Origenis GmbH

Origenis GmbH is a privately-held German biopharmaceutical company
developing brain-penetrating highly selective small molecule medicines
and diagnostics for a variety of neurodegenerative and neuroinflammatory
diseases. Origenis leverages its unique capabilities in drug design,
compound synthesis and characterization to engineer a continuous stream
of proprietary IP-protected new chemical entities capable of permeating
the blood-brain barrier. Origenis’ approach has been validated by
multiple partners resulting in a significant IP and R&D portfolio that
ensures strong patent protection. For more information, visit

About Expansion Therapeutics

Expansion Therapeutics is a drug discovery and development company
pursuing the vast potential of small molecule medicines for RNA-mediated
diseases. Based on exclusive worldwide rights to groundbreaking research
from the laboratory of Matthew D. Disney, Ph.D., at The Scripps Research
Institute, Expansion has assembled the intellectual property, know-how,
and proprietary enabling technologies and tools necessary to facilitate
the creation of potent and specific small molecule binders of RNA.
Through this unique platform, Expansion is building a portfolio of novel
RNA-targeted drug candidates with activity across a broad number of
disease indications. The company’s initial development focus is on
therapies for patients with expansion repeat diseases who currently have
limited and unsatisfactory treatment options. Expansion is based in San
Diego, California and Jupiter, Florida. For more information, visit


Michael Almstetter, CEO
Tel: +49 (0)89 780 16 760

Dr. Peter Seufer-Wasserthal, CBO
Tel: +49 (0)89 780 16 760

Source: Origenis GmbH

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