What Scenic Biotech does

Scenic Biotech B.V. is a privately held Dutch biotech founded in 2017 in Amsterdam that develops disease-modifying therapies for rare neuro- and metabolic genetic disorders through a proprietary approach called modifier therapy. Rather than targeting the primary disease-causing mutation, the company identifies and acts on compensatory "modifier genes" that can neutralize or bypass disease impact. The core technology is Cell-Seq™, a functional genomics platform that uses haploid human cells and gene-trap mutagenesis to systematically screen for modifier genes, generate high-resolution disease maps, and validate novel drug targets. Scenic has built a proprietary data warehouse of modifier genes across more than a dozen diseases and has published extensively in Nature and Science between 2015 and 2025.

The company's pipeline includes wholly owned small-molecule and antisense oligonucleotide programs. The lead asset, SC6177, is a brain-penetrant PLA2G15 inhibitor in IND-enabling studies for Batten Disease, Niemann Pick Type C, Frontotemporal Lobar Degeneration, and (as of 2026) Parkinson's disease via the Michael J. Fox Foundation LITE program; first-in-human trials are targeted for 2027. Additional programs include a Galactokinase (GalK) inhibitor for Galactosemia and an ABHD18 mitochondrial modifier (ASO modality) for Barth Syndrome and cardiomyopathy. Multiple undisclosed neurometabolic programs target indications including X-ALD, HSAN1, and MacTel2.

Scenic operates a dual business model: in-house therapeutic development for long-term clinical and commercial value, and Cell-Seq platform licensing to major pharmaceutical partners (Genentech since 2020, Bristol Myers Squibb since 2024 with two expansions, Alnylam since 2025, and Ono Pharmaceutical since 2026) for upfront payments, research funding, and milestone-based revenue. The company is venture-backed (Eir Ventures, BioMedPartners, Vesalius Biocapital, Inkef, BioGeneration Ventures, Oxford Science Enterprises) with a $31M Series A closed in March 2022 and currently operates with 11–50 employees across Amsterdam headquarters and a New York office.

Name

Scenic Biotech

Legal name

Scenic Biotech B.V.

Website

https://scenicbiotech.com

Company type

Private

Founded year

2017

Operating status

Operating

Headcount range

11–50 employees

Short description

Scenic Biotech is a Dutch development-stage biotech that uses its proprietary Cell-Seq haploid-cell functional genomics platform to identify modifier genes and develop first-in-class small-molecule and ASO therapies for rare neuro- and metabolic genetic diseases, licensing the platform to major pharmaceutical partners.

Ownership category

akta.pro rank

Product category

Rare Disease Therapeutics

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Scientific Research and Development Services (5417)

SIC

Biological Products, (No Disgnostic Substances) (2836), Pharmaceutical Preparations (2834), Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Target Identification & Validation Platforms (omics-driven, chemoproteomics, genetic validation) (HLAAAIAI)

akta.pro secondary industries

Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA), Rare Neurology & Neurodegenerative Disorder Therapies (HLAIAIAD)

Keywords

• Functional genomics platform
• Modifier gene discovery
• Rare disease therapeutics
• Small molecule inhibitors
• Drug discovery services

Headquarters

HQ city

Amsterdam

HQ country

Netherlands

HQ region

Europe

Offices2 records

Markets served

GTM type

B2B

Offering type

Services

Cost components

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

Revenue model

1. Platform Licensing & Collaborations: Scenic Biotech licenses its Cell-Seq platform to major pharmaceutical companies (Genentech, Bristol Myers Squibb, Alnylam, Ono Pharmaceutical) for target discovery, pathway elucidation, and indication expansion. Revenue is generated through upfront payments, research funding, and milestone payments contingent on achievement of research, development, and commercial milestones.
2. In-house Therapeutic Development: Scenic develops wholly owned small molecule inhibitor programs (PLA2G15, GalK, ABHD18, and undisclosed targets) targeting neuro- and metabolic diseases. Revenue would be generated through clinical development and eventual commercialization, or through out-licensing of therapeutic assets to pharma partners.
3. Series A Funding: The company raised $31 million in Series A funding in March 2022, led by Eir Ventures, BioMedPartners, and Vesalius Biocapital, to support its transition into a development-stage biotech and advance its pipeline and collaborations.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels5 records

Core offering

Scenic Biotech develops disease-modifying therapies for rare neuro- and metabolic diseases using its proprietary Cell-Seq™ functional genomics platform, which systematically identifies modifier genes—compensatory genes that counteract disease-causing mutations—via haploid human cell screening and gene-trap mutagenesis. The company licenses this platform to major pharmaceutical companies (Genentech, Bristol Myers Squibb, Alnylam, Ono Pharmaceutical) for target discovery while independently developing a pipeline of first-in-class small molecule and ASO therapeutics, including lead candidate SC6177 (a brain-penetrant PLA2G15 inhibitor for neurodegenerative lysosomal diseases).

Product overview

Scenic Biotech is a neurometabolic company developing disease-modifying therapies based on its proprietary Cell-Seq functional genomics platform. The company's core offering centers on modifier therapy—a radically new approach that acts on compensatory genes to neutralize disease impact rather than targeting primary mutations. The Cell-Seq platform uses haploid human cells and gene-trap mutagenesis to map disease pathways and identify novel drug targets. The lead product, SC6177, is a brain-penetrant small molecule inhibitor targeting PLA2G15 for lysosomal restoration across neurodegenerative diseases. Additional pipeline programs include Galactokinase (GalK) inhibitors for Galactosemia, ABHD18 mitochondrial modifiers for cardiomyopathy, and multiple undisclosed neurometabolic targets. The company operates a fully owned pipeline complemented by strategic collaborations with Genentech, Bristol Myers Squibb, Alnylam, and Ono Pharmaceutical.

Differentiator

Problem solved

Functional benefit

Products and services

• Cell-Seq™ Functional Genomics Platform A proprietary functional genomics discovery platform that uses haploid human cells (carrying only a single copy of every gene) and gene-trap mutagenesis to systematically identify modifier genes—compensatory genes that counteract the effect of disease-causing genes. The platform uses fluorescent labeling, FACS isolation, and DNA sequencing to generate high-resolution disease maps. It is licensed to pharmaceutical companies (Genentech, Bristol Myers Squibb, Alnylam, Ono Pharmaceutical) for target discovery, pathway elucidation, and indication expansion.
• SC6177 (PLA2G15 Inhibitor Program) A brain-penetrant small molecule inhibitor targeting PLA2G15, a lysosomal phospholipase A2 that degrades BMP (bis(monoacylglycero)phosphate), a lipid central to lysosomal function. Designed to elevate endogenous BMP levels and restore lysosomal function in neurodegenerative diseases. Being developed for Batten Disease, Niemann Pick Type C (NPC), Frontotemporal Lobar Degeneration (FTLD), and Parkinson's disease. In IND-enabling studies with first-in-human trials targeted for 2027.
• Galactokinase (GalK) Inhibitor Program A small-molecule allosteric covalent inhibitor program targeting Galactokinase (GalK) for the treatment of Galactosemia, a rare inherited disorder of carbohydrate metabolism that causes neurological and psychiatric deficits. Preclinical studies currently being initiated.
• ABHD18 Mitochondrial Modifier Program An antisense oligonucleotide (ASO) program targeting ABHD18, a novel therapeutic target identified through Cell-Seq as a key regulator of cardiolipin biosynthesis and mitochondrial integrity. ABHD18 loss was shown to overcome severe disease characteristics caused by BTHS-associated TAZ gene, achieving full suppression of all Barth Syndrome disease hallmarks in preclinical models. Published in Nature 2025.
• Undisclosed Neurometabolic Target Programs Multiple genetically validated undisclosed therapeutic targets for neurometabolic diseases, including programs targeting X-linked adrenoleukodystrophy (X-ALD, affecting the nervous system and adrenal glands), HSAN1 (inherited peripheral neuropathy), and MacTel2 (macular telangiectasia type 2, an eye disease affecting the macula).

Quantifiable outcome

• PLA2G15 inhibition significantly improves disease course in NPC mouse models, with improved neurodegeneration, reduced spleen and liver damage, improved neurological symptoms, and increased survival
• +2 more outcomes

Segments2 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature6 records

Partnerships

Ten partnerships are on record, tiered core and supporting.

• Michael J. Fox Foundation (MJFF)coreStrategic or Co-development Partner · 11 June 2026Scenic Biotech joined the Michael J. Fox Foundation's LRRK2 Investigative Therapeutics Exchange (LITE) program to evaluate its first-in-class PLA2G15 inhibitor program as a disease-modifying approach for Parkinson's disease, including patients with LRRK2 mutations. The collaboration leverages LITE's LRRK2 biology expertise, biomarker infrastructure, and validated preclinical models developed at the University of Dundee to generate translational data supporting future clinical development. PLA2G15 inhibitors are designed to elevate endogenous BMP levels and restore lysosomal function, addressing a core driver of neurodegeneration linked to impaired glucocerebrosidase activity in genetic forms of Parkinson's disease.
• University of Dundee (MRC Protein Phosphorylation Unit)supportingStrategic or Co-development Partner · 11 June 2026University of Dundee, through the MJFF LITE program, provides validated preclinical models and biomarker expertise to test Scenic's PLA2G15 inhibitors in the context of LRRK2-linked Parkinson's disease. Dario Alessi, PhD, Director of the MRC Protein Phosphorylation Unit, serves as LITE Principal Investigator.
• Bristol Myers SquibbcoreTechnology or Integration · 26 January 2026Scenic further expanded its platform collaboration with Bristol Myers Squibb, reaching a milestone and expanding the scope to identify novel target biology. The partnership leverages Scenic's Cell-Seq platform to unlock underlying genetic interactions of cellular pathways for undisclosed Bristol Myers Squibb targets, facilitating indication selection and expansion and accelerating drug development.
• Ono PharmaceuticalcoreTechnology or Integration · 7 January 2026Scenic entered a license and research agreement with Ono Pharmaceutical to utilize Scenic's Cell-Seq platform for the discovery of novel therapeutic targets. This marks Scenic's fourth Cell-Seq collaboration with a major pharmaceutical company, joining ongoing multi-year agreements with Genentech, Bristol Myers Squibb, and Alnylam.
• The Hospital for Sick Children (SickKids), TorontosupportingStrategic or Co-development Partner · 3 September 2025Scenic collaborated with Dr. Jason Moffat's team at The Hospital for Sick Children (SickKids) in Toronto on a study identifying ABHD18 as a modifier gene for Barth Syndrome. The study was published in Nature 2025 and identified ABHD18 as a novel therapeutic target, with deactivation of ABHD18 found to rescue mitochondrial defects and achieve full suppression of Barth Syndrome disease hallmarks in preclinical models.
• Alnylam PharmaceuticalscoreTechnology or Integration · 26 August 2025Scenic entered a license and research agreement with Alnylam Pharmaceuticals to leverage its Cell-Seq platform for discovering novel targets for RNAi therapeutics. This collaboration marks Scenic's third Cell-Seq collaboration with major US biotech and pharmaceutical companies, following ongoing multi-year agreements with Genentech and Bristol Myers Squibb. The collaboration harnesses Cell-Seq's capacity to deliver genetic insights that drive novel therapeutic discovery and development.
• Bristol Myers Squibb (Extended Collaboration)coreTechnology or Integration · 12 June 2025Scenic extended its research collaboration with Bristol Myers Squibb for the second time, working to identify target biology for indication selection and expansion. The growth of this partnership highlights the unique power of the Cell-Seq platform to unlock underlying genetic interactions of cellular pathways for novel drug targets.
• Stanford University (Dr. Monther Abu-Remaileh Lab)supportingStrategic or Co-development Partner · 7 May 2025Scenic collaborated with Dr. Monther Abu-Remaileh's team at Stanford University on the discovery and validation of PLA2G15 as a drug target for neuro- and metabolic diseases. The research was published in Nature 2025 and demonstrated that inhibiting PLA2G15 can significantly improve disease course in NPC mouse models, with relevance to Batten Disease, Niemann Pick Type C, and Frontotemporal Lobar Degeneration.
• Bristol Myers Squibb (Initial Collaboration)coreTechnology or Integration · 30 April 2024Scenic entered a research collaboration with Bristol Myers Squibb to accelerate the development of Bristol Myers Squibb's drug targets by identifying target biology for indication selection and expansion. Scenic received an upfront payment and potential additional payments contingent upon achievement of research, development, and commercial milestones.
• GenentechcoreTechnology or Integration · 1 January 2020Scenic Biotech collaborated with Genentech to discover modifier genes to tackle complex diseases. Scenic utilizes its Cell-Seq platform to identify drug targets in multiple therapeutic areas of Genentech's interest. This multi-year partnership highlights mutual commitment to innovation and belief in the transformative potential of genetic research. Announced as Scenic's first major pharma collaboration.

Scale indicators5 records

Recent moves7 records

Expansion highlights6 records

Direct peers

• Maze Therapeutics: Pioneer in modifier-gene-driven drug discovery, also founded by Charles Homcy and using functional genomics to identify compensatory genes in genetic disease. Closest conceptual comparator to Scenic's modifier-therapy thesis.
• Denali Therapeutics: Focused on neurodegeneration and lysosomal biology, with a lead program (tividenofusp alfa) for CLN2/Batten Disease. Directly competes with Scenic's PLA2G15 program in lysosomal indications and shares brain-penetrant therapeutic modality.

Broad incumbents

• Recursion Pharmaceuticals: AI/ML-enabled drug discovery platform using high-throughput biology for novel target identification. Competes with Cell-Seq in supplying pharma partners with functional-genomics-derived targets, though at a much larger scale.
• BridgeBio Pharma: Genetic-disease-focused biotech with multiple rare-disease programs including in cardiology and metabolism. Comparable as a rare-disease therapeutics platform that licenses and develops genetically validated targets.
• BioMarin Pharmaceutical: Established rare-disease biotech with marketed therapies in Batten Disease (Brineura) and other lysosomal disorders. Direct overlap with Scenic's PLA2G15 indications and represents both competitor and potential out-licensing partner.

Emerging players

• Crinetics Pharmaceuticals: Rare-endocrine-disease biotech with approved therapy (Palsonify). Comparable as a development-stage rare-disease company using genetic/biological insights to develop small-molecule therapies with pharma-partnered economics.
• Insitro: ML-enabled functional-genomics platform identifying novel targets through induced pluripotent stem cells. Competes with Cell-Seq in supplying pharma partners with genetically validated targets, with a partnership model overlapping with Scenic's.
• Verve Therapeutics: Genetic-medicine company using genomics-derived insights to develop cardiovascular therapies, with pharma partnerships (e.g., Lilly). Comparable as a platform company monetizing genetic insights through partnerships and in-house pipeline.

Others

• Haplogen Genomics: Co-founded by Scenic co-founder Sebastian Nijman; uses haploid genetic screens for target discovery. Closely related platform technology peer founded on the same scientific foundation as Cell-Seq.

Regional players

• MaaT Pharma: European clinical-stage biotech focused on microbiome-related therapies for severe diseases. Comparable as a European rare-disease biotech in similar development stage and partnership model, though different therapeutic modality.

Market position

Strengths4 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/scenic-biotech

Revenue estimate

Valuation estimate

Number of profiles

Profiles15 records

Funding overview

Funding rounds3 records

Investors8 records

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M&A

Investments

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Frequently asked questions about Scenic Biotech