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 (AAV9-BAG3) (RCSI): Preclinical phase, anticipated IND mid-2022. AAV-BAG3 for heart failure: Research/Discovery phase. AAV-BAG3 for an undisclosed CV indication and undisclosed CNS indication: Both Research/Discovery phase. Renovacor owns commercial rights to all.

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
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