What Casma Therapeutics does

Casma Therapeutics is a privately held, preclinical-stage biotechnology company founded in 2018 and headquartered in Cambridge, Massachusetts, that develops novel therapeutics by harnessing autophagy — the cell's natural lysosomal degradation pathway. The company's proprietary PHLYT™ (Phagosome-Lysosome Targeting) platform uses heterobifunctional small-molecule chemistry to initiate target-specific autophagic degradation of disease-related cellular components, including organelles, protein aggregates, and large signaling complexes that are beyond the capacity of conventional ubiquitin-proteasome or E3 ligase-based degraders. Casma's pipeline is anchored by CSM-101, a first-in-class orally bioavailable, CNS-penetrant small-molecule agonist of TRPML1 (a critical lysosomal ion channel) nominated in June 2025 as its first development candidate for Gaucher's disease patients with Parkinson's disease, with expansion potential into GBA-associated and broader Parkinson's populations. Additional discovery-stage programs target MYD88 mutant lymphoma via the PHLYT™ degrader platform, muscular dystrophy through TRPML1-mediated plasma membrane repair, and lysosomal storage disorders.

The company has raised approximately $154.5 million in cumulative equity financing across a $58.5M Series A (2018, Third Rock Ventures), $50M Series B (2020, led by The Column Group), and $46M Series C (2022, with Amgen Ventures, Astellas, Eisai, and others), plus $7.6M in non-dilutive grant funding from The Michael J. Fox Foundation awarded in March 2026. Casma has no approved products and generates no commercial revenue; its primary customers in the future will be patients with neurodegenerative diseases, oncology indications, and lysosomal storage disorders, with go-to-market likely via partnership with large pharmaceutical companies or independent commercialization following clinical development. The scientific founding team comprises globally recognized autophagy researchers (Andrea Ballabio, Beth Levine, James Hurley, Herbert 'Skip' Virgin), and an IND filing for CSM-101 is planned for the first half of 2026.

Name

Casma Therapeutics

Legal name

Casma Therapeutics, Inc.

Website

https://casmatx.com

Company type

Private

Founded year

2018

Operating status

Operating

Headcount range

11–50 employees

Short description

Casma Therapeutics is a preclinical-stage biotechnology company developing autophagy-based therapeutics via its proprietary PHLYT™ platform, with lead asset CSM-101 — a first-in-class TRPML1 agonist for Parkinson's and Gaucher's disease.

Ownership category

akta.pro rank

Product category

Autophagy-based Therapeutics / Targeted Protein Degradation

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Pharmaceutical Preparations (2834), Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Cheminformatics & Molecular Modeling / CADD (HLAGAJAE)

akta.pro secondary industries

Cell Therapy Enabling Technologies (gene editing, cell engineering, delivery methods) (HLAAABAL), mRNA Therapeutics (vaccines, protein replacement, immuno-oncology) (HLAAADAA)

Keywords

• Autophagy drug discovery
• Targeted protein degradation
• Lysosomal therapeutics
• Small molecule pharmaceuticals
• Neurodegeneration therapeutics

Headquarters

HQ city

Cambridge

HQ country

United States

HQ region

North America

Offices2 records

Markets served

GTM type

B2B

Offering type

Services

Cost components

Technology or R&D, Personnel, Operations, Infrastructure, Marketing or Sales

Revenue model

1. Biopharmaceutical Development: Pre-revenue biotechnology company developing novel therapeutics based on autophagy pathway. No approved products or commercial revenue yet. Revenue generation expected upon successful clinical development and regulatory approval of drug candidates.

Go-to-market motion2 records

Marketing channels6 records

Core offering

Casma Therapeutics is a preclinical-stage biotechnology company developing novel therapeutics that harness the autophagy pathway to selectively degrade disease-causing cellular components beyond the capacity of the ubiquitin-proteasome system. Its proprietary PHLYT™ (Phagosome-Lysosome Targeting) platform uses heterobifunctional chemistry to recruit autophagy regulators to disease targets, enabling degradation of large and complex substrates such as organelles, protein aggregates, and signaling complexes. The lead clinical-stage asset is CSM-101, a first-in-class, orally bioavailable, CNS-penetrant small-molecule TRPML1 agonist for Gaucher's disease patients with Parkinson's disease.

Product overview

Casma Therapeutics is a biotechnology company developing novel cellular degradation approaches based on the autophagy pathway. The company's core offering is the PHLYT™ (Phagosome-Lysosome Targeting) platform, which enables selective autophagic degradation of disease targets beyond the capacity of traditional ubiquitin-proteasome approaches. The company's lead product is CSM-101, a first-in-class TRPML1 agonist for Parkinson's disease and Gaucher's disease. The pipeline includes multiple PHLYT™ programs in oncology and Parkinson's disease, as well as an mTOR-independent autophagy agonist program, all at discovery or preclinical stages.

Differentiator

Problem solved

Functional benefit

Products and services

• PHLYT™ (Phagosome-Lysosome Targeting) Platform Proprietary heterobifunctional chemistry platform that recruits autophagy regulators to disease targets to drive autophagosome biogenesis around the substrate, enabling selective degradation of organelles, protein aggregates, and large signaling complexes beyond the capacity of E3 ligase or proteasomal degraders. Applied across oncology, inflammation, neurodegeneration, and metabolic disorders.
• CSM-101 (TRPML1 Agonist) Lead clinical-stage asset: a first-in-class, orally bioavailable, CNS-penetrant small molecule agonist of TRPML1, a critical lysosomal ion channel. Designed to restore lysosomal function in patients with Gaucher's disease with Parkinson's disease (GD-PD), with potential expansion to GBA-PD and idiopathic Parkinson's disease.
• TRPML1 Agonist CNS Program Pipeline program targeting the TRPML1 lysosomal ion channel to enhance lysosomal efficiency and modulate dynamics in CNS diseases where lysosomal function is compromised, including neurodegeneration.
• MYD88 Mutant Lymphoma Program (Autophagy Degrader) Autophagy degrader program using the PHLYT™ platform to develop degraders targeting disease drivers in MYD88 mutant lymphoma.

Quantifiable outcome

• CSM-101 demonstrated high CNS exposure, potent activity across clinically relevant models, significant reductions in toxic lipid levels, decreased neuroinflammation, and improved survival in multiple Gaucher's disease models
• +2 more outcomes

Segments4 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Two partnerships are on record, tiered supporting and core.

• Vipergen ApSsupportingTechnology or Integration · 1 December 2020Drug discovery agreement where Vipergen applies its proprietary high-fidelity DNA-encoded library (DEL) technology platform to discover novel small-molecule drug leads against undisclosed Casma discovery targets. Casma retains exclusive rights to globally commercialize any products resulting from the collaboration.
• Fondazione Telethon / TIGEM (Telethon Institute of Genetics and Medicine)coreStrategic or Co-development Partner · 11 September 2018Research collaboration to develop new therapies for lysosomal storage disorders and common disorders such as neurodegeneration. The partnership combines Casma's autophagy biology and drug discovery expertise with TIGEM's clinical experience and insights into rare genetic diseases. TIGEM is led by Casma founder Andrea Ballabio, M.D., director of TIGEM.

Scale indicators3 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• Arvinas: Arvinas is a clinical-stage targeted protein degradation company pioneering PROTAC-based bifunctional degraders. It is the closest functional peer to Casma, sharing a heterobifunctional degrader chemistry paradigm and a goal of eliminating disease-driving proteins, although Casma routes degradation through the lysosome via autophagy rather than the proteasome via E3 ligases.
• Kymera Therapeutics: Kymera is a clinical-stage biotech developing heterobifunctional small-molecule degraders leveraging E3 ligase chemistry. It competes with Casma in the broader targeted-degradation space and shares a similar platform-plus-pipeline business model aimed at oncology and immunology indications.
• C4 Therapeutics: C4 Therapeutics develops targeted protein degraders using its TORPEDO platform, a heterobifunctional small-molecule approach conceptually adjacent to Casma's PHLYT™. Both companies pursue degrader-based therapies across oncology indications.
• Lycia Therapeutics: Lycia is pioneering LYTAC (lysosome-targeting chimera) degraders that route disease targets to the lysosome for degradation. Casma's autophagy-based PHLYT™ platform shares the lysosomal-degradation mechanism, making Lycia a close mechanistic peer in the emerging 'extracellular and organelle-targeted degradation' category.
• Monte Rosa Therapeutics: Monte Rosa develops molecular glue degraders (MGDs) to selectively degrade disease-driving proteins via the ubiquitin-proteasome system. It is a relevant peer in the broader targeted-degradation field with a comparable platform-company business model, though differentiated from Casma's autophagy/lysosomal mechanism.

Emerging players

• Denali Therapeutics: Denali is a clinical-stage biotech developing therapeutics for neurodegenerative diseases, with active programs in Parkinson's and lysosomal storage disorders. It overlaps with Casma's CNS-penetrant TRPML1 agonist approach (CSM-101) for GBA-PD and broader PD, though Denali uses enzyme replacement and antibody-based modalities rather than autophagy induction.
• Prothena Corporation: Prothena is a clinical-stage biotech developing protein-immunotherapy approaches for neurodegenerative diseases, including alpha-synuclein-targeted therapies for Parkinson's. It addresses the same GBA-PD and idiopathic PD populations targeted by CSM-101, representing a competing disease-modifying approach.
• Foghorn Therapeutics: Foghorn is a clinical-stage biotech using a chromatin biology platform to develop small-molecule therapies in oncology. Like Casma, Foghorn is a platform-driven biotech leveraging proprietary biology (chromatin regulatory complexes vs. autophagy machinery) to address previously undruggable disease targets.
• Navitor Pharmaceuticals: Navitor developed small molecules targeting the mTOR pathway, which is mechanistically upstream of autophagy regulation. While its chemistry is different, Navitor's focus on mTOR/autophagy signaling overlap with Casma's mTOR-independent autophagy agonist program and broader autophagy-biology thesis.

Broad incumbents

• BioMarin Pharmaceutical: BioMarin is an established global biopharmaceutical company with multiple approved enzyme-replacement therapies for lysosomal storage disorders. Casma's autophagy-enhancer approach to LSDs (clearance of accumulated substrates) represents an alternative modality to BioMarin's enzyme replacement, and BioMarin is the dominant incumbent partner/buyer in this therapeutic space.

Market position

Strengths1 record

Weaknesses5 records

Competitive moat3 records

Key risks7 records

Key highlights7 records

Customer concentration

• Xx.com/casmatx
• LinkedInlinkedin.com/company/casma-tx

Revenue estimate

Valuation estimate

Number of profiles

Profiles10 records

Funding overview

Funding rounds5 records

Investors13 records

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M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Casma Therapeutics