What Skyhawk Therapeutics does

Skyhawk Therapeutics is a clinical-stage biotechnology company developing small molecule therapeutics that modulate RNA expression to address historically undruggable disease targets. The company's proprietary SKYSTAR platform integrates four complementary components — SKYSTAR (target assessment system combining bioinformatic, structural, and computational biology data), SKYLIBRARY (custom-built compound libraries), SKYSEQ (proprietary multiplex screening), and SKYAI (machine learning toolset) — to discover and advance orally bioavailable small molecules that act on RNA upstream of protein expression. The platform has been used to screen more than 700 RNA targets for pharma partners.

The company's lead asset, SKY-0515, is an oral small molecule RNA splicing modulator for Huntington's disease that targets two pathogenic mechanisms simultaneously — mutant huntingtin (mHTT) protein reduction and PMS1-mediated somatic CAG repeat expansion. SKY-0515 has progressed through Phase 1 trials (demonstrating up to 72% HTT mRNA reduction in healthy volunteers and 69% mHTT reduction at 12 months in patients) and is now enrolling a global Phase 2/3 FALCON-HD pivotal trial across 8 countries. Additional pipeline assets include SKY-1214 (an IND-ready FANCL/FANCI RNA splicing modulator for multiple myeloma and non-Hodgkin's lymphoma) and four preclinical rare neurological disease programs (SKY-0925, SKY-2100, SKY-4100, SKY-4200).

Skyhawk generates revenue through upfront payments, development and regulatory milestones, and tiered royalties from pharma collaborations, with over $550 million in upfront payments received to date from nine major partners including Bristol-Myers Squibb, Biogen, Genentech, Sanofi, Takeda, Vertex, Merck & Co., Merck KGaA (deal exceeding $2 billion), and Ipsen (deal worth up to $1.8 billion). The company has raised over $600 million in equity financing, including a $133 million round in September 2021 led by Fidelity. Headquartered in Waltham, Massachusetts with European operations in Basel, Switzerland, Skyhawk is preparing for direct commercial launch of SKY-0515 in Australia via the TGA provisional approval pathway (application submitted March 2026) and targeting major market approvals by 2027, supported by its appointment of a Chief Commercial Officer in March 2026.

Name

Skyhawk Therapeutics

Legal name

Skyhawk Therapeutics, Inc.

Website

https://skyhawktx.com

Company type

Private

Founded year

2018

Operating status

Operating

Headcount range

51–100 employees

Short description

Skyhawk Therapeutics is a clinical-stage biotechnology company developing orally bioavailable small molecule RNA splicing modulators through its proprietary SKYSTAR platform to target historically undruggable proteins in neurodegenerative, oncology, and rare diseases, with lead asset SKY-0515 in Phase 2/3 for Huntington's disease.

Ownership category

akta.pro rank

Product category

RNA-targeting small molecule therapeutics

NAICS

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

SIC

Services-Misc Health & Allied Services, Nec (8090)

akta.pro primary industry

RNA Therapeutics Platforms & Design (sequence design, target ID/validation, AI/bioinformatics) (HLAAADAG)

akta.pro secondary industries

RNA Therapeutics Clinical Development (trial design, endpoints, patient stratification) (HLAAADAH), Rare Neurology & Neurodegenerative Disorder Therapies (HLAIAIAD)

Keywords

• RNA splicing modulators
• Small molecule therapeutics
• Neurology drug discovery
• Targeted oncology therapeutics
• Rare disease pipeline

Headquarters

HQ city

Waltham

HQ country

United States

HQ region

North America

Offices2 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. Pharma Collaboration Payments and Milestones: Skyhawk generates revenue through upfront payments, milestone payments, and tiered royalties from pharma partnerships. Upfront payments from pharma collaborations total over $550 million, with additional potential from milestone payments under collaboration agreements including deals with Merck KGaA ($2B+ total deal value) and Ipsen (up to $1.8B total deal value).
2. Pharmaceutical Product Sales (Future): Once regulatory approvals are obtained, Skyhawk plans to commercialize its proprietary pipeline products, starting with SKY-0515 for Huntington's disease, through direct commercial teams targeting healthcare providers and patients globally.

Go-to-market motion1 record

Distribution channels2 records

Marketing channels5 records

Core offering

Skyhawk Therapeutics discovers and develops oral small molecule drug candidates that modify RNA expression to target historically undruggable proteins implicated in neurodegenerative, oncologic, neuromuscular, rare, and fibrotic diseases. Its clinical-stage pipeline is led by SKY-0515 for Huntington's disease (Phase 2/3 FALCON-HD pivotal trial) and includes SKY-1214 for multiple myeloma and non-Hodgkin's lymphoma (IND-ready), plus four preclinical rare neurology programs. The proprietary SKYSTAR discovery platform integrates SKYSTAR, SKYLIBRARY, SKYSEQ, and SKYAI to generate partnered and wholly-owned assets.

Product overview

Skyhawk Therapeutics is a clinical-stage biotechnology company developing a portfolio of small molecule RNA-modulating drug candidates. The core platform is SKYSTAR, which integrates four components (SKYSTAR target assessment, SKYLIBRARY compound libraries, SKYSEQ multiplex screening, and SKYAI machine learning) to discover RNA-targeting small molecules. Lead product SKY-0515 is in Phase 2/3 pivotal trial for Huntington's disease. Additional programs include SKY-1214 (IND-ready for oncology: multiple myeloma and NHL), SKY-0925, SKY-2100, SKY-4100, and SKY-4200 for rare neurological diseases. The company has collaborations with eight major pharmaceutical partners including BMS, Biogen, Genentech, Ipsen, Merck, Sanofi, Takeda, and Vertex. All drug candidates are small molecules taken orally with demonstrated blood-brain barrier penetration.

Differentiator

Problem solved

Functional benefit

Brands

• SKYSTAR: Proprietary RNA-targeting small molecule drug discovery platform integrating bioinformatics, structural biology, and machine learning
• SKYLIBRARY
• SKYSEQ
• SKYAI

Products and services

• SKY-0515 Oral, once-daily small molecule RNA splicing modulator for early-stage Huntington's disease that simultaneously reduces mutant huntingtin (mHTT) protein and PMS1 mRNA. Currently in Phase 2/3 FALCON-HD pivotal trial with TGA provisional approval pathway eligibility in Australia and broader market entry anticipated by 2027.
• SKY-1214 First-in-class oral FANCL/FANCI RNA splicing modulator for difficult-to-treat multiple myeloma (MM) and non-Hodgkin's lymphoma (NHL) that targets the Fanconi anemia DNA damage repair pathway. IND-ready with completed IND-enabling studies.
• SKY-0925 Preclinical rare neurological disease program targeting an undisclosed indication with significant unmet need and limited therapeutic options. Part of Skyhawk's internal neurology pipeline.
• SKY-2100 Preclinical rare neurological disease program targeting an undisclosed indication with significant unmet need and limited therapeutic options.
• SKY-4100 Preclinical rare neurological disease program targeting an undisclosed indication with significant unmet need and limited therapeutic options.
• SKY-4200 Preclinical rare neurological disease program targeting an undisclosed indication with significant unmet need and limited therapeutic options.
• SKYSTAR Platform Proprietary RNA-targeting small molecule drug discovery platform integrating target assessment (SKYSTAR), custom compound libraries (SKYLIBRARY), multiplex screening (SKYSEQ), and machine learning (SKYAI). Used to support partnered collaborations with Bristol-Myers Squibb, Biogen, Genentech, Sanofi, Takeda, Vertex, Merck (MSD), Ipsen, and Merck KGaA.

Quantifiable outcome

• 69% reduction in mutant huntingtin protein (mHTT) at 9mg dose at 12 months in Phase 1/2 trial
• +5 more outcomes

Named customers1 record

Segments1 record

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

AI capability2 records

Feature4 records

Partnerships

Nine partnerships are on record, tiered major and core.

• Merck KGaA, Darmstadt, GermanymajorStrategic or Co-development Partner · 18 August 2024Skyhawk entered an option agreement granting Merck KGaA exclusive global rights to drug candidates pursued under the collaboration upon option exercise. Total deal value exceeds $2 billion including upfront and milestone payments, along with royalties on future sales. Skyhawk uses its SKYSTAR platform to identify small molecule candidates directed at specific RNA targets designated by Merck KGaA in neurological indications. Following option exercise, Merck KGaA assumes responsibility for further development and commercialization.
• IpsenmajorStrategic or Co-development Partner · 22 April 2024Ipsen entered an exclusive worldwide collaboration to discover and develop novel small molecules modulating RNA for rare neurological diseases. Deal worth up to $1.8 billion including upfront and milestone payments plus tiered royalties. Ipsen receives option to acquire exclusive global rights to two candidates following development candidate nomination, then assumes all further development and commercialization responsibilities. Ipsen brings neuroscience expertise in movement disorders.
• Bristol-Myers Squibb (BMS)coreTechnology or IntegrationBMS collaboration leverages Skyhawk's novel SKYSTAR platform to target challenging diseases across various therapeutic areas including neurodegenerative disease, autoimmune disease, and oncology. Upfront payments from pharma collaborations total over $550 million.
• BiogencoreTechnology or IntegrationBiogen collaboration leverages Skyhawk's novel SKYSTAR platform to target challenging diseases across various therapeutic areas. Upfront payments from pharma collaborations total over $550 million.
• Genentech (Roche)coreTechnology or IntegrationGenentech collaboration leverages Skyhawk's novel SKYSTAR platform to target challenging diseases across various therapeutic areas. Upfront payments from pharma collaborations total over $550 million.
• SanoficoreTechnology or IntegrationSanofi collaboration leverages Skyhawk's novel SKYSTAR platform to target challenging diseases across various therapeutic areas. Upfront payments from pharma collaborations total over $550 million.
• TakedacoreTechnology or IntegrationTakeda collaboration leverages Skyhawk's novel SKYSTAR platform to target challenging diseases across various therapeutic areas. Upfront payments from pharma collaborations total over $550 million.
• VertexcoreTechnology or IntegrationVertex collaboration leverages Skyhawk's novel SKYSTAR platform to target challenging diseases across various therapeutic areas. Upfront payments from pharma collaborations total over $550 million.
• Merck & Co.coreTechnology or IntegrationMerck & Co. collaboration leverages Skyhawk's novel SKYSTAR platform to target challenging diseases across various therapeutic areas. Upfront payments from pharma collaborations total over $550 million.

Scale indicators10 records

Recent moves7 records

Expansion highlights6 records

Direct peers

• PTC Therapeutics: PTC Therapeutics is the most direct comparable — a commercial-stage company that originated the RNA splicing modulator field with risdiplam (Evrysdi), co-invented by Skyhawk co-founder Sergey Paushkin. PTC operates the same small-molecule RNA splicing modality and shares Skyhawk's CNS/splicing therapeutic focus.
• Wave Life Sciences: Wave is a leading direct competitor in Huntington's disease, developing allele-selective antisense oligonucleotides (WVE-003) targeting the same mHTT protein. Wave is the closest therapeutic rival to SKY-0515 in HD and shares the rare neurology/splicing-modulating therapeutic strategy.
• uniQure: uniQure is the principal gene therapy competitor in Huntington's disease with AMT-130 (AAV5-miHTT), currently pursuing accelerated approval. AMT-130 represents the primary modality-based competitive threat to SKY-0515 in the HD market.

Emerging players

• Arrakis Therapeutics: Arrakis is a clinical-stage biotech developing direct RNA-targeting small molecules (rSM), operating a platform conceptually similar to Skyhawk's SKYSTAR. It represents the most direct emerging-platform competitor in RNA-binding small molecule discovery.
• Expansion Therapeutics: Expansion Therapeutics is developing small molecules that target repeat-expansion RNAs in diseases including Huntington's and myotonic dystrophy. It overlaps with Skyhawk on the rare neurology repeat-expansion biology (PMS1/CAG expansion) that SKY-0515 addresses.

Broad incumbents

• Ionis Pharmaceuticals: Ionis is the established leader in antisense oligonucleotide (ASO) therapeutics with a broad neurology pipeline. Ionis developed tominersen (with Roche) for Huntington's disease and Spinraza for SMA, making it a direct therapeutic-area incumbent adjacent to Skyhawk's CNS focus.
• Alnylam Pharmaceuticals: Alnylam is the commercial leader in RNA interference (siRNA) therapeutics. While its modality differs from Skyhawk's small-molecule approach, Alnylam shares the RNA-targeting therapeutic category and competes for pharma partnership attention in RNA-targeted drug development.
• BioMarin Pharmaceutical: BioMarin is a commercial-stage rare-disease biotech with CNS programs and proven specialty distribution economics. It is comparable as a model for how Skyhawk's HD and rare-neuro franchise could scale commercially once SKY-0515 launches.
• Moderna: Moderna is the leading commercial mRNA therapeutics company, providing a parallel RNA-modality comparable as Skyhawk scales its pipeline. While the therapeutic modality (mRNA versus RNA-binding small molecules) differs, both companies operate within the broader RNA therapeutics category.
• Sage Therapeutics (acquired by Biogen): Sage Therapeutics (now a Biogen subsidiary) developed CNS rare-disease therapies including Zurzuvae, with a comparable specialty neurology commercial profile. It is relevant as a peer for the commercial-stage rare neurology model Skyhawk is preparing to enter with SKY-0515.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xx.com/Skyhawk_Tx
• LinkedInlinkedin.com/company/skyhawk-therapeutics

Revenue estimate

Valuation estimate

Number of profiles

Profiles6 records

Subsidiaries1 record

Funding overview

Funding rounds5 records

Investors11 records

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M&A

Investments

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Frequently asked questions about Skyhawk Therapeutics