OUR MISSION

To bring the advantages of small molecule medicines to the unmet needs of hereditary disorders.

A Genetic Disease Focus

We focus on genetic disorders for several reasons.

First, there is still a significant need for therapies in these areas. Most genetic disease patients are only treated for symptoms, allowing the disease to progress and not improving patients’ lives long-term.

Second, unlike many diseases treated with conventional therapies, genetic diseases
have a known cause, and directly treating the cause affords the opportunity to reverse
the disease.

Third, the ability to identify patients by genetic testing allows for targeted therapy administration, ensuring that only those who will benefit from the treatment receive it. This greatly improves the success rate in clinical trials. Additionally, it reduces healthcare system costs by avoiding expenditure on ineffective treatments. Most importantly, it transforms the diagnosis from a negative prognosis into a source of hope.

A Platform that Increases Speed, Lowers Costs and Gives Better Results for Patients

There, we said it: Better, Cheaper, Faster.

Sharp Therapies are Better for Patients

Most genetic diseases are treated with biologics, i.e. protein drugs or gene-based drugs.

Sharp Therapies (as small molecule drugs) will be patients’ first choice therapy over any biologic because:

  1. They are an oral pill rather than an injection, infusion, or administration of a genetic therapy.
  2. Patients appreciate the convenience of obtaining a prescription from a pharmacy and administering the medication at home or elsewhere, without needing to schedule their lives around injections.
  3. Unlike biologics, patients do not run the risk of an allergic reaction that may cause them to halt therapy, or even worse lead to hospitalization.
  4. Unlike genetic therapies, discontinuation of treatment simply means not taking your pill.

Sharp Therapies are Better for Providers and the Healthcare System

  1. They are less expensive (contrast protein and genetic medicines)
  2. Reimbursement is like any other drug in their formulary (contrast gene-based medicines).

Sharp Therapies are “Cheaper” Because They are:

  1. Less expensive to discover
  2. Less expensive to manufacture
  3. Less expensive to prove clinically
  4. Less expensive to bring to market

Sharp Therapies are Faster

  1. We use industrial lab automation to dramatically accelerate discovery.
  2. We use disease models that are more predictive of clinical success and are faster than conventional animal models.
  3. Once in clinical testing we learn far earlier that the therapy is working and therefore worthy of continuing (contrast pharma drug development which typically requires lengthy and expensive trials to prove the clinical hypothesis).
  4. A therapy for genetic disease is often granted accelerated regulatory review.
  5. Genetic disease therapies often gain approval significantly earlier in the review process than conventional therapies meaning faster (and cheaper) development.