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
Bcell lymphomas
ORN-101
Wholly owned
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.
GENETIC DISEASE
Dystrophin
replacement
DMD
Wholly owned
+OrNAgade JV
Orna has achieved non-viral delivery of a large, 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).
VACCINES
SARS-CoV2 spike
COVID-19
OrNAgade JV
Orna is investigating the suitability its oRNATM technology combined with intramuscularly administered immunotropic LNPs for vaccine applications, including for COVID-19. The oRNATM half-life observed in muscle and immune cells, combined with the intramuscular administration of immunotropic lipids, suggests that oRNA-LNP technology may be beneficially applied to vaccine development.
OTHER
Undisclosed
Wholly owned
+OrNAgade JV
Discovery
Preclinical
Clinical
isCARTM
PRODUCT: Anti-CD19 CAR
INDICATION: Bcell lymphomas
PARTNERS: Wholly owned
DISCOVERY
PRECLINICAL
CLINICAL
ORN-101
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.
GENETIC DISEASE
PRODUCT: Dystrophin replacement
INDICATION: DMD
PARTNERS: Wholly owned +OrNAgade JV
DISCOVERY
PRECLINICAL
CLINICAL
Orna has achieved non-viral delivery of a large, 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).
VACCINES
PRODUCT: SARS-CoV2 spike
INDICATION: COVID-19
PARTNERS: OrNAgade JV
DISCOVERY
PRECLINICAL
CLINICAL
Orna is investigating the suitability its oRNATM technology combined with intramuscularly administered immunotropic LNPs for vaccine applications, including for COVID-19. The oRNATM half-life observed in muscle and immune cells, combined with the intramuscular administration of immunotropic lipids, suggests that oRNA-LNP technology may be beneficially applied to vaccine development.
OTHER
PRODUCT: Undisclosed
INDICATION:
PARTNERS: Wholly owned +OrNAgade JV
DISCOVERY
PRECLINICAL
CLINICAL