Harvard Launches Spinout to Address mRNA, CRISPR Challenges

Harvard University recently launched Vesigen Therapeutics, which will address the challenges of delivering RNAi, mRNA, and CRISPR technologies to intracellular targets in specific tissues of interest.

Through a license agreement with Harvard, Vesigen Therapeutics will develop and commercialize drug-delivery technologies that originated in the lab of Quan Lu, professor of environmental genetics and physiology at the Harvard Chan School. 

The company’s scientific advisory board will also include Stephen Haggarty, PhD, of Massachusetts General Hospital, and Thomas Reh, PhD, of the University of Washington.

The new advancement is expected to boost the development of new therapeutics for various conditions in which intracellular is currently a roadblock. The spinout will specifically leverage the capability of “ARMMs,” said Lu in the announcement.

ARMMs or ARRDC1-mediated microvesicles technology, are a class of fusogenic extracellular vesicles that nature evolved to package and deliver communication signals between cells and tissues. ARMMs have the potential to solve major challenges for the biotech industry. 

“We can harness the capability of these vesicles, called ARMMs” — for ARRDC1-mediated microvesicles — “to first package and then deliver therapeutic cargoes to the targeted tissues. That’s the ultimate goal of this, to enable next-generation therapeutics to reach their full potential in combating a wide range of diseases,” explained Lu explained. 

Some of the most promising new therapeutics, such as CRISPR editing, mRNA, or RNAi molecules, are large proteins and nucleic acids that cannot cross the cell membrane without help.

The key innovation from the novel drugs found in Lu’s lab, Harvard explained, comes from the discovery of an unrecognized mechanism in the cell membrane that allows it to accept deliveries without sending them to be destroyed. 

“Many therapeutic modalities would benefit from a drug-delivery mechanism with low immunogenicity, an intrinsic ability to traffic to specific tissues and cell types, and an efficient capability to deliver cargo directly to the cytoplasm of target cells,” said Grant Zimmermann, managing director of business development in Harvard OTD.  

“RNA- or protein-based therapeutic cargo is directly loaded into the ARMM delivery vehicle during biogenesis, streamlining the biological manufacturing process. The innovations from the Lu Lab are extremely promising in these respects, and I’m thrilled to see them enter commercial development.”

Vesigen launched with $28.5 million in Series A investment led by Bayer and Morningside Ventures. Linden Lake Ventures and Alexandria Venture Investments also joined in on the funding.

Vesigen will use the capital raised to build out the ARMMs platform and to advance various therapeutic agents into preclinical and clinical development. 

With the financing, Vesigen named Gerald Chan of Morningside Ventures as chairman, while Stephen Bruso of Morningside Ventures and Juergen Eckhardt and Jak Knowles of Leaps by Bayer have been named to its board of directors. 

“Leaps by Bayer is investing in transformative biotechnologies with the ability to move the paradigm from treatment to cure,” Juergen Eckhardt, MD, head of Leaps by Bayer, said in a separate press release. 

“We believe that Vesigen’s ARMMs technology has the potential to do exactly that, to help enable new curative treatments in a large spectrum of disease areas.”

Bayer explained that Vesigen and its scientific founders have demonstrated a wide range of therapeutic payloads can be packaged in ARMMs, including RNA, protein, and gene-editing systems and functionally delivered intracellulary in vitro and in vivo.

“Vesigen’s engineered extracellular vesicles show versatility and we will be supporting their development in a wide range of indications, including oncology, neurology, ophthalmology, and other localized applications – We look forward to seeing their work translated into a clinical context,” said Gerald Chan, ScD, Morningside Ventures.