Perlara
Perlara, PBC is a precision drug discovery company that uses humanized yeast disease avatars to screen repurposable compounds for inherited metabolic diseases. It partners with patient foundations and academic centers, and advanced its flagship PMM2-CDG program to Phase III under $5M.
- Company typePrivate
- Founded2014
- HeadquartersSan Francisco, United States
- Headcount1–10
- GTM typeB2B
- OfferingServices
What Perlara does
Perlara, PBC is a precision drug discovery company founded in 2014 and headquartered in South San Francisco that develops therapies for ultra-rare inherited metabolic diseases (IMDs). It operates a yeast-first, patient-partnered drug discovery platform called REMIT (Repurposing with Engineered Models of Inherited metabolic disease Targets), which uses humanized yeast disease avatars — primarily Yarrowia lipolytica and Saccharomyces cerevisiae — to screen a standardized library of 8,384 repurposable compounds (sourced from TargetMol) across 34 IMD models covering congenital disorders of glycosylation (PMM2, ALG11, DHDDS), mitochondrial disorders (electron transport chain complexes CI–CV, SURF1, ECHS1), GPI-anchor disorders (PIGN, PIGW), tRNA-synthetase syndromes (NARS1, FARS2, MTAARS2), and others (FOXG1, ADSL). Hits are validated in patient-derived cells before advancing through IND-enabling and registrational trials. The company's flagship program, Maggie's Pearl, advanced epalrestat — a Japanese aldose-reductase inhibitor approved for diabetic neuropathy — from a parent-initiated N-of-1 study to a 38-patient Phase III registrational trial at Mayo Clinic Minnesota in under three years for less than $5M in patient-foundation capital.
All screening data is published through a public data portal (dashboard.perlara.com) before IP filing, an unusual transparency choice that doubles as both credibility-building content and a published working notebook via the Cure Odysseys Substack. The company currently runs 12 active programs with a stated ambition to scale to approximately 600 over the next decade.
Perlara's revenue model is multi-stream but project-based: (1) NIH and SBIR grant funding — most recently a $1.7M Direct-to-Phase-II SBIR from NIH NCATS in November 2025 for inherited mitochondrial disease platform development, (2) patient-foundation capital funding individual PerlQuest/Cure Odyssey programs, (3) out-licensing of IND-enabling-ready assets to pharma and biotech partners (with patient-foundation rights and Cure Odysseys narration access retained), and (4) joint ventures such as Maggie's Pearl. Customer segments are rare disease families and patient foundations (primary), academic research groups (primary), and pharma/biotech licensing partners (secondary). The company is a founder-led Public Benefit Corporation under CEO Ethan Perlstein, with a lean core team of approximately six scientific, clinical, and operations members supported by a decentralized wet lab.
Perlara firmographics
Firmographics- Name
- Perlara
- Legal name
- Perlara, PBC
- Website
- https://perlara.com
- Company type
- Private
- Founded year
- 2014
- Operating status
- Operating
- Headcount range
- 1–10 employees
- Short description
- Perlara, PBC is a precision drug discovery company that uses humanized yeast disease avatars to screen repurposable compounds for inherited metabolic diseases. It partners with patient foundations and academic centers, and advanced its flagship PMM2-CDG program to Phase III under $5M.
- Ownership category
- akta.pro rank
Perlara industry classification
Industry- Product category
- Biotech Drug Discovery
- NAICS
- Scientific Research and Development Services (5417), Pharmaceutical and Medicine Manufacturing (3254)
- SIC
- Biological Products, (No Disgnostic Substances) (2836)
- akta.pro primary industry
- Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA)
- akta.pro secondary industries
- Library & Diversity Generation Platforms (display libraries, DNA-encoded libraries for biologics, variant libraries) (HLAAAIAL), Translational Medicine & Biomarkers (HLAIALAF)
Keywords
Where Perlara is headquartered
LocationHeadquarters
- HQ city
- San Francisco
- HQ country
- United States
- HQ region
- North America
Offices2 records
Markets served
Perlara business model
Business model- GTM type
- B2B
- Offering type
- Services
- Cost components
- Personnel, Technology or R&D, Operations, Marketing or Sales, Supply Chain
Revenue model
- NIH SBIR Grants: Direct-to-Phase-II SBIR grant from NIH NCATS awarded in November 2025 for $1.7M to develop yeast-based drug discovery platforms for inherited mitochondrial diseases, covering electron transport chain models.
- Patient-Foundation Funding: Funding from patient foundations and families (e.g., Maggie's PMM2-CDG Cure, Cure Sanfilippo Foundation, Multiple Sulfatase Deficiency Action Foundation) to support specific PerlQuest/Cure Odyssey programs. Patient-foundation capital supported the Maggie's Pearl Phase III for under $5M.
- Licensing / Out-licensing: Out-licensing of IND-enabling-ready assets to pharma and biotech partners who can advance development and commercialization. Perlara retains patient-foundation rights and Cure Odysseys narration access.
- Maggie's Pearl Joint Venture: Joint venture with a pioneer family, Maggie's Pearl, to develop and commercialize the epalrestat program for PMM2-CDG. Represents the first co-developed asset from the platform.
Go-to-market motion2 records
Distribution channels1 record
Marketing channels6 records
Perlara product offering
Product offeringCore offering
Perlara is a full-stack drug discovery and development company for inherited metabolic diseases. It builds humanized yeast disease avatars, screens a standardized 8,384-compound repurposing library, validates hits in patient-derived cells, advances programs through IND-enabling studies, and out-licenses or co-develops assets (e.g., Maggie's Pearl epalrestat for PMM2-CDG) with pharma, biotech, and patient foundations.
Product overview
Perlara is a full-stack drug discovery and development company operating through an integrated platform centered on the REMIT Engine. The core offering combines yeast-based disease modeling with patient partnership programs, including the flagship Maggie's Pearl (epalrestat for PMM2-CDG, now in Phase III). Supporting infrastructure includes the Data Portal for public screening data access, Cure Odysseys Substack as the working notebook, and the Pipeline tracking active programs across CDGs, mitochondrial disorders, GPI-anchor defects, and tRNA-synthetase syndromes. Historical offerings from the Perlara 2.0 era include Guided Cures consulting services.
Differentiator
Problem solved
Functional benefit
Brands
- REMIT: Repurposing with Engineered Models of Inherited metabolic disease Targets — a four-stage drug discovery engine: build yeast avatar, screen library, validate in patient cells, advance through IND-enabling and trials.
- Cure Odysseys
- Guided Cures™
- PerlQuests
Products and services
- REMIT Engine Repurposing with Engineered Models of Inherited metabolic disease Targets (REMIT): a four-stage drug discovery engine — build the yeast avatar, screen a standardized library, validate in patient cells, advance through IND-enabling and trials. Used by Perlara's scientific team and partners to advance rare metabolic disease programs.
- Maggie's Pearl (epalrestat for PMM2-CDG) Flagship co-developed asset and joint venture with a pioneer family; a parent-initiated N-of-1 study of epalrestat (a Japanese diabetic-neuropathy drug) for PMM2-CDG that reached a 38-patient Phase III at Mayo Clinic Minnesota in under three years for less than $5M in patient-foundation capital.
- Public Data Portal Public interactive dashboard providing access to Perlara's yeast screening data across 34 IMD models and 8,384 TargetMol compounds, with gene/disease overview, compound x gene heatmaps, compound explorer, dual-condition pairs, and per-compound fingerprints. De-identified data is published publicly before IP filing.
- Cure Odysseys Substack Public Substack notebook narrating each Perlara program in real time, serving as both a working lab notebook and a community engagement channel documenting the family, yeast model, hit list, N-of-1 protocol, and what fails.
- Guided Cures™ Consulting Services Modality-agnostic virtual consulting and model-agnostic drug repurposing services offered during the Perlara 2.0 era to families and scientists seeking to advance treatments and cures for rare diseases.
- IMD Pipeline (12 active programs targeting CDGs, mitochondrial disorders, GPI-anchor defects, tRNA-synthetase syndromes) Active inherited metabolic disease programs covering congenital disorders of glycosylation (PMM2, ALG11, DHDDS, GMPPA), mitochondrial electron transport chain disorders (SURF1, ECHS1), GPI-anchor disorders (PIGN, PIGW), tRNA-synthetase syndromes (NARS1, FARS2, MTAARS2), and other ultra-rare metabolic biology; out-licensed or co-developed with patient foundations, academic medical centers, and pharma partners.
Quantifiable outcome
- Phase III reached in under 3 years for under $5M patient-foundation capital (Maggie's Pearl/epalrestat for PMM2-CDG)
- +3 more outcomes
Companies that use Perlara
Customer profileNamed customers12 records
Segments3 records
Ideal customer profiles3 records
Perlara technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature5 records
Perlara partnerships and signals
Strategic signalPartnerships
14 partnerships are on record, tiered major, minor and flagship.
- CDG UKmajorSupporting an international clinical trial for DHDDS-related congenital neurodegenerative disease involving 12 patients over one year, using patient-derived mini-brain organoids and nicotinamide mononucleotide (NMN) as a potential therapeutic. Some families have already reported improvements from NMN supplementation.
- Harvard / Undiagnosed Diseases NetworkmajorCollaboration on GNAO1 neurodevelopmental disorder drug discovery using patient-derived yeast avatars. Development of variant-specific GNAO1 models (including A221D) in collaboration with the Undiagnosed Diseases Network and Harvard Medical School.
- Radical InvestmentsmajorLeigh Syndrome PerlQuest partnership with Radical Investments. A new treatment for Leigh syndrome is noted to have broader implications for aging, sports medicine, traumatic brain injury, and neurodegeneration.
- Rambam Medical CentermajorAPOL1 kidney disease PerlQuest partnership with Rambam Medical Center. APOL1 genetic risk affects approximately 100 million people worldwide.
- Phelan-McDermid Syndrome FoundationminorPerlQuest exploration for Phelan-McDermid Syndrome (PMS), including engagement at the 2018 International Family Conference. PMS involves SHANK3 and related neurodevelopmental pathways potentially addressable through Perlara's platform.
- Cedars-Sinai Medical CentermajorParallel collaboration with Cedars-Sinai Medical Center established through the Mission: Cure partnership. Small molecules discovered in Perlara's yeast screens are advanced directly to testing in an artificial pancreas system using cells derived from pancreatitis patients.
- NovartismajorCollaboration with Novartis focused on lysosomal diseases, including developing worm and fly models for Multiple Sulfatase Deficiency (ML4) and Niemann-Pick Type C (NPC) using a curated bioactive collection ('MoA Box') for high-throughput screening.
- University of Notre Dame (Warren Center for Drug Discovery)majorCollaboration on screening platform for glycogen storage diseases including Cori Disease (GSD III) and Pompe Disease (GSD II). The Warren Center provides academic infrastructure for fly model development and drug screening for these conditions.
- Mission: CuremajorPancreatitis PerlQuest partnership to develop yeast models of 27 CFTR patient variants and screen for small molecule therapeutics. Mission: Cure also established a parallel collaboration with Cedars-Sinai Medical Center so that yeast-discovered hits can be advanced to testing in patient-derived cell systems.
- Multiple Sulfatase Deficiency Action FoundationmajorMSD PerlQuest partnership for Multiple Sulfatase Deficiency, including fly model development and phenotypic screening. Built as part of the broader Novartis lysosomal diseases collaboration.
- Cure Sanfilippo FoundationmajorMPSIII PerlQuest partnership for Sanfilippo syndrome (MPSIIIA and MPSIIIB), involving nematode and fly disease models and compound screening programs.
- Wylder Nation FoundationmajorPerlQuest partnership for Niemann-Pick Type A. Perlara identified tool compounds, repurposable drugs, and novel chemotypes that rescue worm and fly disease models.
- Grace Science FoundationmajorNGLY1 PerlQuest partnership focused on yeast and fly disease models and drug repurposing screens for NGLY1 Deficiency. The partnership was among Perlara's earliest patient foundation collaborations.
- Maggie's PMM2-CDG Cure (Maggie's Pearl)flagshipFlagship parent-initiated N-of-1 study that became a joint venture and the template for the Perlara 3.0 model. Epalrestat (a Japanese diabetic-neuropathy drug) advanced from compassionate use to a 38-patient Phase III at Mayo Clinic Minnesota in under 3 years for under $5M. The first Cure Odyssey and first joint venture with a pioneer family.
Scale indicators6 records
Recent moves6 records
Expansion highlights6 records
Perlara competitors and assessment
Company assessmentBroad incumbents
- Travere Therapeutics: Rare disease biopharmaceutical company with approved therapies and pipeline focused on metabolic disorders, including lysosomal storage diseases that overlap with Perlara's glycosylation and GPI-anchor programs. Note: Travere was an early Perlara investor (2014), suggesting historic strategic alignment.
- BioMarin Pharmaceutical: Established rare disease biopharmaceutical company with multiple approved enzyme replacement therapies and small molecule drugs for inherited metabolic disorders including CLN2, MPS, and PKU. Represents the scaled commercial endpoint of the patient-foundation-to-registrational-trial model Perlara aspires to.
- Alexion / AstraZeneca Rare Disease: AstraZeneca's rare disease unit focused on rare metabolic and hematologic disorders, including approved therapies for lysosomal storage diseases and hypophosphatasia. A likely strategic acquirer or licensing partner for Perlara's IND-ready metabolic disease assets at scale.
- BridgeBio Pharma: Genetic disease-focused biopharmaceutical company developing therapeutics for rare and underserved conditions. Overlap with Perlara's inherited metabolic disease focus (e.g., metabolic disorders), but BridgeBio operates at significantly larger scale with broad pipeline across multiple rare disease modalities.
- Ultragenyx Pharmaceutical: Clinical-stage biopharmaceutical company focused exclusively on rare and ultra-rare genetic diseases, including multiple inherited metabolic disorders. Comparable patient-foundation relationships and ultra-rare focus, but operates as a full integrated biotech with approved products versus Perlara's platform-discovery model.
- Pompe Therapeutics (Amicus): Rare disease biotech with approved and pipeline therapies for inherited metabolic disorders including Fabry disease and Pompe disease (GSD II), which Perlara also has a screening collaboration on with Notre Dame. Strong comparability on disease area, though Amicus is significantly larger and integrated.
Direct peers
- Maze Therapeutics: Genetic disease drug discovery company focused on translating insights from genetic variation into oral small molecule therapies. Shares Perlara's genetic-disease-first approach and patient-foundation partnership orientation, though Maze focuses on common variant-informed targets rather than ultra-rare inherited metabolic disease.
- Recursion Pharmaceuticals: Tech-enabled drug discovery platform combining high-throughput biology with machine learning. Like Perlara, it pursues repurposing and novel targets using model organism data, though at much larger scale and across broader therapeutic areas; their shared 'biology-first, platform-native' ethos makes them a direct comparator.
Others
- Perlara PBC itself lists a University of Notre Dame partnership on Pompe/GSD III: Academic rare disease screening centers and contract research organizations supporting patient-derived disease modeling and drug screening represent adjacent ecosystem partners rather than direct competitors, but compete for the same foundation funding and patient-foundation engagement relationships.
Emerging players
- Cydan Development: Orphan drug accelerator that identifies and de-risks therapeutic candidates for rare diseases before spinning out standalone companies. Conceptually similar to Perlara's patient-foundation incubation model, though Cydan operates across modalities and at venture-scale rather than yeast-based discovery.
Market position
Strengths5 records
Weaknesses5 records
Competitive moat6 records
Key risks5 records
Key highlights6 records
Customer concentration
Perlara social profiles
Digital presencePerlara financial estimates
Financial estimateRevenue estimate
Valuation estimate
Perlara leadership team
Management profileNumber of profiles
Profiles6 records
Perlara funding detail
Funding detailFunding overview
Funding rounds7 records
Investors6 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Perlara M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Perlara
What does Perlara do?
Perlara is a full-stack drug discovery and development company for inherited metabolic diseases. It builds humanized yeast disease avatars, screens a standardized 8,384-compound repurposing library, validates hits in patient-derived cells, advances programs through IND-enabling studies, and out-licenses or co-develops assets (e.g., Maggie's Pearl epalrestat for PMM2-CDG) with pharma, biotech, and patient foundations.
Is Perlara a public or private company?
Perlara is a private company. It is classified as founder individual operated bootstrapped and is currently operating.
When was Perlara founded?
Perlara was founded in 2014. It employs 1 to 10 people.
Where is Perlara based?
Perlara is headquartered in San Francisco, United States, in the North America region.
How does Perlara make money?
Four revenue lines are on record. NIH SBIR Grants are the primary driver. The others are patient-Foundation Funding, licensing / Out-licensing and maggie's Pearl Joint Venture.
Who are Perlara's main competitors?
Broad incumbents on record are Travere Therapeutics, BioMarin Pharmaceutical, Alexion / AstraZeneca Rare Disease, BridgeBio Pharma, Ultragenyx Pharmaceutical and Pompe Therapeutics (Amicus). Direct peers are Maze Therapeutics and Recursion Pharmaceuticals. Perlara PBC itself lists a University of Notre Dame partnership on Pompe/GSD III is listed as an others. Cydan Development is listed as an emerging player.
Does Perlara have an API?
No public API is recorded for Perlara.
What industry is Perlara in?
Perlara's product category is Biotech Drug Discovery. Its primary akta.pro industry code is HLAIAIAA, Genetic & Genomic Rare Disease Therapeutics, with a secondary code of HLAAAIAL, Library & Diversity Generation Platforms (display libraries, DNA-encoded libraries for biologics, variant libraries). Its NAICS code is 5417 and its SIC code is 2836.