DEVELOPING PRECISION THERAPIES FOR RARE DISEASES
4X FASTER AT 50X LOWER COST

Rarefy Therapeutics LLC is a biopharma incubator based on a proprietary platform technology LADR4 to expeditiously advance therapies into first-in-human studies for rare diseases. We have a particular focus on therapeutics to address inherited disorders of energy production, a class of genetic disorders collectively known as Primary Mitochondrial Diseases (PMD).

LINKER-AIDED DRUG REPURPOSE, REPOSITION, REPROFILE, RESCUE (LADR4).

LADR4 is an industry first, platform technology that allows for repurposing of generic drugs by creating New Chemical Entities (NCEs) with composition of matter (COM) patent claims. These NCEs have improved physico-chemical properties and PK/PD profile, but do not trigger the need for a 505(b)1 development path. This provides a one-of-a-kind strategy that:

  • Makes repurposing investable by generating a full 20 year composition of matter claim based on NCEs
  • Shortens time to approval
  • Lowers cost to approval
  • Increases probability of success
  • Provides a significant return for investors

LADR4 addresses the urgent need to develop therapies for individuals living today with rare disease, by reducing both the time and cost to obtain approval.

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Structure

Rarefy Therapeutics intends to advance therapeutic programs for First-in-Human clinical trials. A unique structure will be employed for each program, where a specific “Project Corporation” will be created and managed by a Governing Board that provides cost-effective access to strategic and executive leadership. The Governing Board will have representation from Rarefy Therapeutics and investors, and will be empowered to act as an independent group with requisite authority and responsibility. Following the successful completion of First-in-Human studies (e.g. Investigator Initiated Study), the Governing Board will actuate the process of creating a NewCo (having a substantially increased pre-money valuation) and determine the funding, financing, and development strategy and plans to help raise the investment needed to advance the Project through USFDA approval.

Value

Rarefy Therapeutics is focused on creating value for both rare disease patients and investors. LADR4 is the unique, patented approach that will enable realization of this vision.

With improved probability of success, reduced time to success, and lower cost of success – Rarefy Therapeutics offers a novel approach to develop a range of effective therapies for rare disease patients.

Founders

Sundeep Dugar, PhD

Serial entrepreneur with 35+ years in drug discovery & development and co-inventor of Zetia® and Vytorin®.

Marni J. Falk, MD

Physician-scientist and Clinical Geneticist with 24 years’ experience in rare genetic disease, preclinical disease modeling, and clinical trials, as well as internationally recognized thought leader in Mitochondrial Medicine.

Contact Us

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