Lysinia Therapeutics

HOMEDeveloping a treatment for Glutaric Aciduria Type 1 (GA1),
a rare genetic disease affecting children.

EMAIL: [email protected]

team

Four founders combining academic rigor with practical drug discovery experience.Click to view:

Edward A Kesicki   /   Robert J DeVita   /   Sander M Houten   /   Michael B Lazarus
CEO - Founder                Founder                     Founder                         Founder       

The Science and Opportunity

Our drug molecule blocks the pathway that leads to accumulation
of toxic glutaric acid in cells, a hallmark of the disease.We have:
- Lead small-molecule inhibitor of the pathway - potent
- ~$8M funding to date, mostly from NIH/NICHD
- X-ray structure-enabled program
- Proof-of-concept in rodent models - simple biomarker readout
- Clear path to clinic
- Provisional patents filed 2026
- Eligible for FDA "Priority Review Voucher" on drug approval (worth $150M)
- Working with the Clinic for Special Children near Lancaster, PA, a facility
   with an emphasis on GA1 patient care

Some background and contact info

Glutaric Aciduria Type 1 (GA1) :
- Inborn error of metabolism - autosomal recessive
- Diagnosed at birth in the US
~1000 GA1 cases in the US (30-40 births/year)
- Clustered:  Amish / Indigenous groups
- Neurotoxic / developmental disorder
- Most vulnerable up to age 6:  A single fever can
   cause irreversible brain injury
- Current treatment:  Low-lysine diet and carnitine
   supplementation.  Sub-optimal

Contact:      [email protected]

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