Perlara

The first biotech public benefit corporation (est. 2014) now developing medicines for inherited metabolic diseases (MITO, CDG).

[Perlara @PerlaraPBC]

Introducing Perlara 3.0 -- one mission, three eras: https://t.co/fyNyv0Fkfz

We’re now a full-stack medicines company for inherited metabolic diseases: congenital disorders of glycosylation, mitochondrial diseases, and the long tail of ultra-rare metabolic biology.

[Kristin Kantautas @kkantaut]

Rare disease drug development has a math problem.

One drug. One disease. $1B+. 10+ years.

That math doesn't work for 1500+ rare metabolic diseases. The proof: 80% still have no treatment.

At @PerlaraPBC & Cure SRD5A3 I’ve spent 6 years discovering and developing medicines for rare diseases. The TradBio model doesn't work for rare.

My takeaway: many rare metabolic diseases break down at the same hubs in metabolism.

Target the hub. Treat many diseases with a single medicine.

Today I'm announcing @Metabolisbio. A biotech built with my co-founder @eperlste to develop keystone medicines: a new class of treatments for rare metabolic diseases.

We discover naturally occurring metabolites and develop them into medicines that can treat many rare diseases.

We also run AI-powered at-home patient studies. Faster data. Lower cost. No traveling to a trial site.

Our first program is NMN for DHDDS-CDG. A pediatric neurodegenerative disease.

NMN has already shown it can slow disease progression in patients. Read the preprint: https://t.co/KVD7uAq1DQ

We're raising a pre-seed round. If you believe rare disease is the proving ground for the next generation of metabolic medicines let's talk.

https://t.co/oh4bvl7YcI

[Ethan Perlstein 1-to-N @eperlste]

📣Attention AIxBIO companies looking for lab-based verification data/loops

@PerlaraPBC has quietly amassed a proprietary genotype-chemotype drug screening atlas for inherited metabolic diseases

Want to develop novel medicines for underserved patient communities? Let's talk!

[Perlara @PerlaraPBC]

Introducing Perlara 3.0 -- one mission, three eras: https://t.co/fyNyv0Fkfz

We’re now a full-stack medicines company for inherited metabolic diseases: congenital disorders of glycosylation, mitochondrial diseases, and the long tail of ultra-rare metabolic biology.

[Perlara @PerlaraPBC]

Be sure to check out our Pipeline (https://t.co/7PF1ESIAxe) and Data Portal (https://t.co/Zsik1XXKvE) to learn more about our 30+ programs!

[Perlara @PerlaraPBC]

Introducing Perlara 3.0 -- one mission, three eras: https://t.co/fyNyv0Fkfz

We’re now a full-stack medicines company for inherited metabolic diseases: congenital disorders of glycosylation, mitochondrial diseases, and the long tail of ultra-rare metabolic biology.

[Perlara @PerlaraPBC]

We've built a cell-state map of inherited metabolic disease pharmacology comprised of 34 disease models x 8,384 compounds.

The big theme is that rescue mechanisms recur by cell state, not by clinical or genetic labels. The dataset supports a cell-biological framework with four major axes:

1. Mitochondrial output / biomass demand
2. Cytosolic protein-economy / biosynthetic supply
3. ER-glycan / secretory lipid-linked flux
4. IMM architecture / respiratory substate

We'll have more to say on our Substack Cure Odysseys soon..

[Perlara @PerlaraPBC]

Perlara 3.0 site launching soon

[Perlara @PerlaraPBC]

We’ve been quietly building Perlara 3.0, the third evolution of the company that will focus on developing novel medicines for inherited metabolic diseases.

A new site, including an AI-powered dashboard summarizing the screening results for 37 diseases, is coming soon!

We’ll debut the dashboard on Friday April 24 at the @CDGCAREorg conference in Orlando.

[Perlara @PerlaraPBC]

The evolution of biotech company

Perlara 1.0 = B2B platform as a service

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Perlara 2.0 = DTC platform as a service

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Perlara 3.0 = DTC drug developer

[Perlara @PerlaraPBC]

⚡️BIG NEWS⚡️

We were awarded a $1.7M SBIR grant to scale up a yeast-powered mitochondrial disease pipeline!

This represents a major milestone/turning point for the company.

It’s the beginning of Perlara 3.0 with a focus on developing medicines for inherited metabolic diseases.

[Perlara @PerlaraPBC]

@hiranhpatel 🙏

[Perlara @PerlaraPBC]

⚡️BIG NEWS⚡️

We were awarded a $1.7M SBIR grant to scale up a yeast-powered mitochondrial disease pipeline!

This represents a major milestone/turning point for the company.

It’s the beginning of Perlara 3.0 with a focus on developing medicines for inherited metabolic diseases.

[Perlara @PerlaraPBC]

Thank you to the Perlara core team, our investors, and to everyone who has stood by us on this long journey, which just feels like it’s just getting started after 11 years.

[Perlara @PerlaraPBC]

Enter Perlara 3.0

Time to focus on bringing a medicine to market starting with mitochondrial diseases like Leigh Syndrome.

We’re also pursuing Congenital Disorders of Glycosylation (CDGs) as a complementary therapeutic area, all under the banner of inherited metabolic diseases.

[Anne Carpenter, PhD @DrAnneCarpenter]

@fulop_dan @PerlaraPBC Perlara absolutely is interested in using many screening setups to connect drugs to diseases! This is a type they currently don’t use, protein localization in cultured human cells, but could. We are discussing in DMs actually whether they’d adopt the project, maybe STTR 😄