Perlara PBC awarded $1.7M SBIR grant to fund yeast-powered drug discovery for mitochondrial diseases

Perlara PBC will focus on inherited mitochondrial diseases involving deficiencies in the electron transport chain (Complexes I to V)

ALAMEDA, CA, UNITED STATES, November 5, 2025 /EINPresswire.com/ -- Perlara PBC, the world’s first biotech public benefit corporation with a mission to develop medicines for rare genetic diseases, in particular inherited metabolic diseases (IMDs), announced that the company was awarded a Direct-to-Phase-2 SBIR grant (REMIT) to expand its mitochondrial diseases drug discovery platform using the non-standard yeast model system Yarrowia lipolytica.

Perlara PBC is building an IMD-focused drug discovery platform starting with mitochondrial diseases caused by deficiencies in any one of hundreds of evolutionarily conserved genes. This approach expands upon foundational work performed in collaboration with cure-focused patient foundations such as Cure Mito and CDG CARE. The REMIT grant allows Perlara PBC to deprecate direct-to-family drug repurposing as a service model and focus instead on developing novel medicines for families affected by inherited metabolic diseases.

The REMIT platform approach takes advantage of pharmacological deficiency models of each complex of the electron transport chain: Complexes I-V. Promising preclinical drug candidates will be validated in patient-derived models and then evaluated in decentralized N-of-few clinical studies. The goal is to identify “platforms in pill,” or rational mitochondrial cocktails that would benefit patients across myriad mitochondrial disease genotypes.

"This funding marks a turning point in the 11-year history of the company and the beginning of Perlara 3.0," said Ethan O. Perlstein, Ph.D., founder and CEO of Perlara PBC. "We are grateful to the scores of patient partners with whom we’ve built in the trenches over the last decade. Working with pioneer families on N-of-1 cases has taught us which processes are scalable from 1 to N. We will apply those hard-fought lessons to make mitochondrial medicines, and this grant represents the down payment."

Follow the medicine-making journey on Perlara’s Substack Cure Odysseys.

About Perlara PBC:

Established in February 2014, Perlara PBC is a rare disease biotech company focused on developing medicines for inherited metabolic diseases, which represent 1 in 10 rare diseases. From 2014 to 2019, Perlara PBC’s multi-species screening platform was partnered with biopharma and patient foundations, resulting in a joint venture with a rare disease family called Maggie’s Pearl. Over the past four years, Perlara PBC has operated as a consultancy and drug repurposing as a service provider.

More at www.perlara.com

Ethan Perlstein
Perlara PBC
ethan@perlara.com
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