Skip to main content
U.S. flag

An official website of the United States government

NIA Small Business Showcase: Juvena Therapeutics, Inc.

Banner signifying OSBR handbook

Tissue degenerates with age and in the context of multiple rare and chronic diseases, leading to life-debilitating illnesses, many with no cure and with major unmet need. Tissue degeneration is caused in part by the loss of homeostatic protein signaling, leading to stem cell exhaustion, impaired intercellular communication, increased inflammation and fibrosis, and reduced tissue repair and regeneration.

Juvena logo

Engineered protein-based therapeutics promote tissue regeneration and repair to treat chronic and rare diseases.

Juvena’s lead program will advance a protein therapy for a rare muscle-wasting disease, myotonic dystrophy type 1 (DM1), with an orphan drug status and a more than $40 billion total addressable market (TAM). This enables accelerated clinical and drug approval by the U.S. Food and Drug Administration (FDA) and thus validation of Juvena’s platform and pipeline. Juvena recognizes the looming health care crisis and urgent need to solve age-related and rare degenerative muscle diseases with unmet medical needs, with costs rising in the United States to more than $300 billion annually.

For Juvena’s lead program for DM1, the median U.S. pricing for orphan drug biologics is $225,000 annually per person. Assuming enrollment of 25% of the U.S. and European Union (EU) market share, the company anticipates more than $2 billion annual U.S. and EU revenue and more than $25 billion over the lifetime of the program. The upper limit is $300,000 annually per person, with an upper potential for a serviceable available market (SAM) of $8 billion annually in unmet need.

Juvena is building a computationally driven platform to map secreted proteins to unmet medical needs by mining the secretomes of stem cells, enabling de novo protein drug discovery and development for tractable chronic and rare diseases. Its platform enables the company to build a compounding database of biologics with disease-modifying potential. There are major pharmaceutical companies that are interested in exploring the therapeutic potential of secreted proteins but that lack systematic methods and tools for cost- and time-effective screening of proteins. Other platform companies targeting mechanisms of aging are repurposing drugs or investigational new drugs (INDs) that did not succeed for primary indications, while Juvena is developing novel biologic drug candidates. Other biologics companies are focused on identifying signaling pathways and proteins by studying the effects of aged and young blood circulation to promote tissue homeostasis or drive pathogenesis. Juvena is unique in mining stem cell secretomes for their therapeutic potential. Other companies focused on targeting mechanisms of aging are developing different modalities, such as mRNA and small molecules, to target senescence, or telomeres. Juvena is uniquely focused on modulating signaling pathways systemically and locally that promote tissue regeneration by restoring homeostatic signaling and promoting endogenous stem cell function, reduction in inflammation and fibrosis, improved mitochondrial function and cellular viability, and other disease-modifying phenotypes by restoring healthy intercellular communication. Other companies in the regenerative medicine space are developing gene therapies that have a complementary modality or are focused on cell transplantation therapies. Stem cell transplantations often have led to transient benefits due to the stem cells secretome’s paracrine and endocrine signaling effects, but these benefits do not last, as the cells often do not survive long after transplantation. Juvena is instead focused on mining the secretomes of stem cells to map the soluble fraction and their secreted proteins to unmet medical needs and develop those substances as injectable biologics that can promote tissue-specific regeneration.

Juvena’s lead drug candidate for DM1 is a biologic, a fusion protein that will be administered subcutaneously and act as an endocrine therapy to promote muscle survival, regeneration, strength, function, and improved metabolism by restoring Akt signaling and other muscle regenerative signaling pathways to healthier levels. To Juvena’s knowledge, JUV-161 is the only protein-based therapeutic being developed for DM1. Other companies are developing gene therapies, small molecules, and antibody-oligonucleotide conjugate antisense therapies. Biologics have established regulatory costs; chemistry, manufacturing, and controls (CMC) costs; and costs of goods sold (COGS). The value of the unique mechanism of action of JUV-161 for DM1 is supported by multiple key opinion leaders. Juvena sees effective muscle tissue delivery, and the company’s drug uniquely improves muscle regeneration and strength, targeting a major hallmark of DM1.

Company Milestones


Scientific — Platform

  • 2018: Completed first secretome library and initiated hit screening for disease-modifying factors that could promote muscle regeneration
  • 2019: Developed predictive models for protein-disease association (PQNET)
  • 2019: Developed machine learning based applications to accelerate in vitro and in vivo screening
  • 2020: U.S. Patent 10,821,155 issued: “heparin-associated polypeptides and uses thereof”
  • 2022: Successfully developed mouse model of DM1
  • 2023: Advance three new molecular entities (NMEs) to lead optimization, validating discovery platform
  • 2023: Complete characterization of four secretomes, initiate new therapeutic area programs, and expand knowledge base

Scientific and Clinical — Pipeline

  • 2021: Awarded NIH Small Business Innovation Research (SBIR) funding for neuromuscular lead optimization
  • 2021: Awarded $3.9 million in California Institute of Regenerative Medicine (CIRM) support for pre-IND activities of neuromuscular lead asset
  • 2022: Developmental candidate nominated for lead program
  • 2022: Expanded candidate discovery programs to four therapeutic areas with several novel hits generated


  • 2017: Juvena Therapeutics founded
  • 2018: Closed $4.3 million seed funding
  • 2019: Closed $1.5 million simple agreement for future equity (SAFE) note financing
  • 2020: Closed $1 million SAFE note financing
  • 2021: CIRM awarded Juvena $3.9 million TRAN1 grant to advance JUV-161 for DM1; Juvena secured $5.7 million SAFE note financing
  • 2022: Closed Series A funding (as yet unannounced)

Financial Overview

Juvena has raised $43 million in venture capital funding and $4.2 million in grant funding from CIRM and NIH. The company currently is seeking $5 million to $10 million through venture capital, ideally by the end of 2022. Juvena’s goal is to raise Series B funding in 2025, after hitting a clinical-stage asset inflection point. The company is also gearing up to apply to several NIH and CIRM grants that will enable lead optimization and preclinical development of novel lead assets.

Intellectual Property (IP)

Juvena has issued and pending IP in the U.S. and major global pharmaceutical markets on the composition of its engineered lead candidates, methods of use for clinical treatment of an array of neuromuscular and musculoskeletal diseases for those assets, methods for producing screening libraries from complex protein secretomes, methods of use for clinical treatment of an array of neuromuscular and musculoskeletal diseases for an instance of such a library, and a machine learning platform for computationally predicting and experimentally vetting the therapeutic potential of components of candidate therapeutic protein libraries.

Product Development and Regulatory Strategy

Juvena’s strategy is to develop a clinical-stage pipeline of biologics that are novel ligands and receptor disease-modifying mechanisms targeting rare and/or degenerative diseases that can be primarily first in class or, opportunistically, best in class for major unmet medical needs. We are doing this by leveraging and expanding our platform JuvNET’s coverage of secretomes, disease/organ/tissue mapping, and in vitro and in vivo models to identify candidate leads across multiple therapeutic areas.

With DM1 as the initial clinical target for our lead program, Juvena’s regulatory strategy aims to secure an orphan drug designation to enable financial incentives and the potential for seven years of market exclusivity after approval. Given the major unmet medical need for DM1, Juvena will also seek a fast-track review process to facilitate the development and expedite the review of our lead and future programs.

Commercialization Strategy

Juvena’s strategy is to build a pipeline of biologics for rare and chronic diseases that we can advance toward approval through pharma licensing, co-development partnerships, and/or directly ourselves through grant and venture capital funding. We will advance some drug assets, particularly for rare orphan diseases, to the clinic with the support of NIH and CIRM funding while focusing partnership deals on leads for larger indications. We expect early-stage revenue to be generated from pharma licensing and co-development of biologics in our pipeline for global distribution and from entering platform discovery deals with major pharma and biotech companies for organ- or indication-specific protein drug discovery. We will explore program partnerships following value-creating milestones, including IND approval and Phase 1/2 clinical data readouts.

Company Details

Juvena Therapeutics, Inc.

Redwood City, CA

Industry: Biotechnology and BioPharma

Management Team:

  • CEO: Hanadie Yousef, Ph.D.
  • CSO: Jeremy O’Connell, Ph.D.
  • Head of Data Science: Edward Moler, Ph.D.

Point of Contact:
Hanadie Yousef
(650) 223-5879

An official website of the National Institutes of Health