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Our Pipeline

A Full-Circle Approach To 
Treating Disease Paint Underline

We are more than the sum of our platforms – with the potential to drive better efficacy and patient outcomes compared to current cell therapy approaches.

Our combined oRNA® and LNP technologies can address the limitations of linear mRNA therapeutics and treat challenging diseases where other modalities have fallen short. With higher protein expression, larger cargo capacity, and lower immunogenicity profile, our therapeutics can go the distance and fill a pipeline with far-reaching effects in oncology, autoimmune, genetic and other diseases.

Pipeline Paint Underline

Delivery
Disease
Approach
Discovery
Development
Candidate
IND
Ph I / II
Partners
panCAR™
Immune
cells
B cell-driven autoimmune diseases
Anti-CD19 in-vivo
CAR
In 2024
By 2026
B cell
malignancies
Anti-CD19 / Anti-BCMA
in-vivo CAR
In 2024
By 2026
Simnova logo

Orna’s in vivo CAR platform (panCAR™) combines oRNA® technology with our proprietary LNPs to create therapies that can modify immune cells within a patient, no lymphodepletion or waiting required. Unlike existing ex vivo CAR-T or CAR-NK therapies, our panCAR™ program generates CAR-T, CAR-NK and CAR-Mac cells at once, and is truly off-the-shelf. This easily redosable format allows for reliable dose control and consistent generation of CAR-bearing immune cells for effective tumor control.

Through our partnership with Simnova Biotherapeutics, we are accessing CAR proteins, rapid early clinical assessment, and the China market for a number of potential oncology therapies.
STEM
Bone
marrow
Sickle cell disease
in-vivo CRISPR editing
In 2024
By 2026
β-thalassemia
in-vivo CRISPR editing
In 2024
By 2026
Our SiTu Editing in the Marrow (STEM)™ platform combines the large cargo capacity of oRNA®, our internally developed novel CRISPR genome editing platform with our proprietary LNPs to create therapies that can potentially correct genetic diseases of the bone marrow (such as sickle-cell disease and beta-thalassemia) within a patient, no lymphodepletion or waiting required, unlike existing ex vivo HSC editing technologies.
Proprietary
Infectious disease
& others
Protective antigens
& other strategies
Merck logo
RNA technology is ideal for vaccine development, as shown by the recent success of multiple mRNA-based vaccine products. In collaboration with Merck, Orna is leveraging the potency and durability of our oRNA® platform coupled with novel LNP delivery insights to develop the next generation of vaccine candidates.
panCAR™ Immune cells
Disease: B cell-driven autoimmune diseases
Approach: Anti-CD19 in-vivo CAR
Discovery/DC
In 2024
Ph I / II
By 2026
Disease: B cell malignancies
Approach: Anti-CD19 / Anti-BCMA in-vivo CAR
Partners: Simnova logo
Discovery/DC
In 2024
Ph I / II
By 2026

Orna’s in vivo CAR platform (panCAR™) combines oRNA® technology with our proprietary LNPs to create therapies that can modify immune cells within a patient, no lymphodepletion or waiting required. Unlike existing ex vivo CAR-T or CAR-NK therapies, our panCAR™ program generates CAR-T, CAR-NK and CAR-Mac cells at once, and is truly off-the-shelf. This easily redosable format allows for reliable dose control and consistent generation of CAR-bearing immune cells for effective tumor control.

Through our partnership with Simnova Biotherapeutics, we are accessing CAR proteins, rapid early clinical assessment, and the China market for a number of potential oncology therapies.
STEM
Bone marrow
Disease: Sickle cell disease
Approach: in-vivo CRISPR editing
Discovery/DC
In 2024
Ph I / II
By 2026
Disease: β-thalassemia
Approach: in-vivo CRISPR editing
Discovery/DC
In 2024
Ph I / II
By 2026
Our SiTu Editing in the Marrow (STEM)™ platform combines the large cargo capacity of oRNA®, our internally developed novel CRISPR genome editing platform with our proprietary LNPs to create therapies that can potentially correct genetic diseases of the bone marrow (such as sickle-cell disease and beta-thalassemia) within a patient, no lymphodepletion or waiting required, unlike existing ex vivo HSC editing technologies.
Proprietary
Disease: Undisclosed
Approach: Protective antigens & other strategies
Partners:
Merck logo
RNA technology is ideal for vaccine development, as shown by the recent success of multiple mRNA-based vaccine products. In collaboration with Merck, Orna is leveraging the potency and durability of our oRNA® platform coupled with novel LNP delivery insights to develop the next generation of vaccine candidates.

Discovery Programs Paint Underline

Orna is continuing to investigate new disease areas where oRNA® and LNP technologies can address unmet need. Our enhanced platform capabilities guide our discovery program work.