Our Pipeline

A NEW WAY TO ADDRESS DISEASE

Orna’s initial focus is on treating challenging diseases where other modalities have fallen short. Orna believes it can apply its oRNATM technology to potentially address the limitations of current immunotherapies by delivering chimeric antigen receptors (CARs) directly to patient’s immune cells within the body (isCARTM therapy). The potential applications of Orna’s technology are far reaching, with a varied pipeline of oncology, genetic disease, and infectious disease targets.

PIPELINE

Product
Indication
Discovery
Preclinical
Clinical
Partners
isCARTM
Anti-CD19 CAR
B-cell lymphomas
ORN-101
Undisclosed
—
Orna’s in situ CAR therapy combines oRNATM technology with proprietary LNPs to create modified immune cells within a patient. This easily redosable format would not require patient lymphodepletion and would allow for reliable dose control, overcoming barriers of existing ex vivo CAR-T therapies without sacrificing efficacy.
GENETIC DISEASE
Dystrophin
replacement
DMD
Undisclosed
—
Orna has achieved non-viral delivery of a full-length dystrophin-encoding RNA in human cells, as well as in vivo delivery of smaller length versions in mouse models. These data are a step on the path to delivering improved gene therapy to patients, such as those with Duchenne Muscular Dystrophy (DMD).
INFECTIOUS DISEASE
Undisclosed
—

Orna, in collaboration with Merck and ReNAgade, is leveraging the potency and durability of oRNATM as well as novel LNP delivery insights to develop next-generation vaccine products.
DISCOVERY PROGRAMS
Undisclosed
—
Orna is investigating the applicability of its oRNATM and LNP technologies across multiple target diseases and enhancing its platform to address unmet needs.
Discovery
Preclinical
Clinical
isCARTM
PRODUCT: Anti-CD19 CAR
INDICATION: B-cell lymphomas
DISCOVERY
PRECLINICAL
CLINICAL
ORN-101
PRODUCT: Undisclosed
INDICATION: —
Undisclosed
PRECLINICAL
CLINICAL
Orna’s in situ CAR therapy combines oRNATM technology with proprietary LNPs to create modified immune cells within a patient. This easily redosable format would not require patient lymphodepletion and would allow for reliable dose control, overcoming barriers of existing ex vivo CAR-T therapies without sacrificing efficacy.
GENETIC DISEASE
PRODUCT: Dystrophin replacement
INDICATION: DMD
PARTNERS:
DISCOVERY
PRECLINICAL
CLINICAL
PRODUCT: Undisclosed
INDICATION: —
Undisclosed
PRECLINICAL
CLINICAL
Orna has achieved non-viral delivery of a full-length dystrophin-encoding RNA in human cells, as well as in vivo delivery of smaller length versions in mouse models. These data are a step on the path to delivering improved gene therapy to patients, such as those with Duchenne Muscular Dystrophy (DMD).
INFECTIOUS DISEASE
PRODUCT: Undisclosed
INDICATION: —
PARTNERS:

DISCOVERY
PRECLINICAL
CLINICAL
Orna, in collaboration with Merck and ReNAgade, is leveraging the potency and durability of oRNATM as well as novel LNP delivery insights to develop next-generation vaccine products.
DISCOVERY PROGRAMS
PRODUCT: Undisclosed
INDICATION: —
DISCOVERY
PRECLINICAL
CLINICAL
Orna is investigating the applicability of its oRNATM and LNP technologies across multiple target diseases and enhancing its platform to address unmet needs.