Our Pipeline
Renovacor is developing a pipeline of BAG3-related gene therapies, with initial programs in BAG3-associated DCM and earlier stage programs targeting BAG3-associated cardiovascular and central nervous system disorders.
REN-001
Renovacor’s lead product candidate, REN-001, is an AAV9-based gene therapy designed to treat BAG3-associated DCM.
REN-001 has undergone extensive preclinical testing. Renovacor intends to file an IND for REN-001 in mid-2022 with Phase I studies to begin thereafter.
REN-001 has been designed with several features to promote safety and efficacy:
Monogenic diseases with well understood biology are ideal targets for AAV GTxs
- Targeting disease with known genetic origin
- BAG3 mutations well-documented as driver in DCM
- Goal is to increase BAG3 levels in DCM subjects
Local (Retrograde Coronary Sinus Infusion, RCSI) delivery allows lower total dose
- Reduces potential for various vector toxicities
- May reduce burden on manufacturing
Utilizes validated AAV9 capsid
- AAV9 currently used in one approved therapy (e.g. Zolgensma)
- AAV9 has demonstrated cardiac tropism
- Has high transduction efficiency
- Non-integrative vector
Non-immunogenic one-time human BAG3 payload
- Therapeutic payload is human BAG3 gene
- DCM patients are haploinsufficient and produce low levels of native BAG3; therefore, the protein is not foreign and should not elicit an immune response