What Skylark Bio does

Skylark Bio is a private, clinical-stage biotechnology company founded in February 2021 and headquartered in Cambridge, Massachusetts. The company develops AAV-based gene therapies intended to address the underlying genetic causes of sensory limitations, with an initial focus on hearing impairment caused by monogenic mutations. Its pipeline includes a GJB2 gene therapy program targeting the most common cause of inherited non-syndromic hearing loss worldwide, and the SKY-PEN pendrin gene therapy program for inherited Pendrin-related hearing loss, with IND-enabling studies underway for the latter. The company also conducts undisclosed CNS research programs that leverage the same vector platform.

The core technology is a proprietary AAV vector platform optimized for targeted micro-dose delivery of therapeutic genes to cells within the contained environment of the cochlea, minimizing systemic exposure. The platform supports both gene addition and CRISPR/Cas9-mediated disruption of dominant mutations, expanding the set of addressable genetic defects. Preclinical validation includes demonstrated AAV transduction efficiency in non-human primates (2018) and hearing restoration across five different mouse models using either modality. The platform originates from co-founder Casey Maguire's AAV bioengineering work at Massachusetts General Hospital and Harvard Medical School, with scientific direction from co-founder David Corey, who heads the Harvard Medical School Center for Hereditary Deafness.

Skylark Bio is pre-revenue and has not yet commercialized a product. Its intended revenue model centers on licensing and royalties for gene therapy assets, with a near-term go-to-market dependent on advancing programs through regulatory milestones (IND-enabling studies and first-in-human trials planned for 2026). A June 2026 strategic CDMO partnership with Forge Biologics secures cGMP AAV manufacturing for clinical supply. The company has raised $60M led by ARCH Venture Partners and operates with a 1–10 person core team augmented by a deep scientific advisory board and academic collaborations with Harvard Medical School and Massachusetts General Hospital. The primary customer base — once commercialized — would be pediatric patients with GJB2- or Pendrin-related hearing loss.

Name

Skylark Bio

Legal name

Skylark Bio, Inc.

Website

https://skylarkbio.com

Company type

Private

Founded year

2021

Operating status

Operating

Headcount range

11–50 employees

Short description

Skylark Bio is a Cambridge, MA-based biotechnology company developing AAV gene therapies targeting genetic causes of hearing loss, with lead programs for GJB2 and pendrin mutations and a first pediatric clinical trial planned for 2026.

Ownership category

akta.pro rank

Product category

Gene Therapy

NAICS

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

SIC

Services-Commercial Physical & Biological Research (8731), Services-Testing Laboratories (8734)

akta.pro primary industry

AAV Vector Gene Therapy Developers (HLAAACAA)

akta.pro secondary industries

Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA), Gene Therapy Regulatory, Pharmacovigilance & Clinical Operations Services (HLAAACAM), Cell Banking & Viral Seed Services (MCB/WCB/VSB) (HLAGAGAD)

Keywords

• Gene therapy development
• Hearing loss treatment
• AAV vector platforms
• Rare disease therapeutics
• Inner ear medicine

Headquarters

HQ city

Cambridge

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Services

Cost components

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

Revenue model

1. Gene Therapy Development: Pre-revenue clinical stage biotech company developing gene therapies for rare genetic diseases. Revenue model not yet established as company is in development stage with first clinical program anticipated in 2026.

Go-to-market motion1 record

Marketing channels3 records

Core offering

Skylark Bio is a clinical-stage biotechnology company developing AAV-based gene therapies that target the underlying genetic causes of hearing impairment. The company uses optimized adeno-associated virus (AAV) vectors to deliver therapeutic genes directly to affected cells in the inner ear (cochlea), with programs addressing GJB2-related and Pendrin-related hereditary deafness, plus undisclosed CNS indications.

Product overview

Skylark Bio is a gene therapy company focused on developing innovative medicines to eliminate sensory limitations, initially targeting hearing impairment. The company's pipeline includes the GJB2 Gene Therapy Program targeting the most common cause of inherited hearing loss, and the SKY-PEN Gene Therapy Program for Pendrin-related hearing loss. Both programs use optimized AAV vectors to deliver therapeutic genes directly to affected cells in the inner ear.

Differentiator

Problem solved

Functional benefit

Products and services

• GJB2 Gene Therapy Program Pre-clinical gene therapy candidate that uses an optimized AAV vector to deliver a functional copy of the GJB2 gene to cells in the cochlea, intended to restore hearing in patients with GJB2-related inherited non-syndromic hearing loss.
• SKY-PEN Gene Therapy Program Gene therapy program for inherited hearing loss caused by pendrin gene mutations, delivered via optimized AAV vectors to inner ear cells. Preclinical data to be presented at ASGCT 2026.

Quantifiable outcome

• Demonstrated AAV transduction efficiency in non-human primates in 2018
• +1 more outcomes

Segments3 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Three partnerships are on record, tiered core.

• Forge BiologicscoreStrategic or Co-development Partner · 16 June 2026Strategic AAV development and cGMP manufacturing partnership supporting Skylark's clinical development. Forge Biologics provides contract development and manufacturing organization (CDMO) services for AAV vector production and clinical supply.
• Massachusetts General HospitalcoreStrategic or Co-development PartnerCo-founder Casey Maguire is Principal Investigator at MGH. Collaboration on AAV vector development and gene therapy approaches for inner ear disorders.
• Harvard Medical SchoolcoreStrategic or Co-development PartnerCo-founder David Corey heads the Harvard Medical School Center for Hereditary Deafness. Scientific collaboration on gene therapy for inner ear and hereditary deafness research.

Scale indicators3 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• Decibel Therapeutics: Gene therapy and antisense oligonucleotide programs for hearing loss and balance disorders; acquired by Regeneron in 2023. Competes head-to-head with Skylark in genetic deafness treatment, including inner ear delivery approaches.
• Akouos: Direct AAV gene therapy competitor targeting genetic hearing loss; co-founder and CSO Gregory Robinson previously served as Akouos CSO prior to its acquisition by Eli Lilly. Most directly comparable company to Skylark in modality, indication, and stage.
• Otonomy: Clinical-stage biotech developing therapeutics for hearing and balance disorders, including otoprotection and inner ear drug delivery. Comparable target indications but uses different modalities (small molecules, sustained-release formulations).
• Sensorion: Clinical-stage French biotech developing gene therapies and small molecules for hearing loss including pediatric genetic deafness. Directly competes with Skylark's lead indications and shares focus on rare monogenic hearing disorders.

Emerging players

• Pipeline Therapeutics: Clinical-stage biotech developing therapeutics for hearing loss and neuroprotection. Targets overlapping indications including genetic and noise-induced hearing loss with small molecule and biologic approaches.
• Frequency Therapeutics: Clinical-stage regenerative medicine company developing small molecule therapies to regenerate sensory hair cells in the inner ear. Addresses hearing loss through a different mechanism but in the same anatomical target and indication space.
• Rinri Therapeutics: UK-based biotech developing cell-based therapies to restore hearing through regeneration of auditory neurons. Overlapping sensory neuroscience platform approach with potential indication overlap.
• Audion Therapeutics: Clinical-stage biotech developing small molecule therapeutics for hearing loss including otic regeneration programs. Targets same genetic and age-related hearing loss indications through a different modality.

Broad incumbents

• REGENXBIO: Established AAV gene therapy platform company with broad pipeline across ophthalmology, CNS, and metabolic diseases. Provides AAV vector technology and manufacturing that is broadly comparable to Skylark's underlying AAV platform, though targeting different indications.

Others

• Forge Biologics: CDMO specializing in AAV development and cGMP manufacturing; strategic partner to Skylark for clinical supply. Ecosystem participant rather than direct competitor, but indicative of comparable gene therapy manufacturing infrastructure.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat4 records

Key risks5 records

Key highlights6 records

Customer concentration

• LinkedInlinkedin.com/company/skylark-bio

Revenue estimate

Valuation estimate

Number of profiles

Profiles10 records

Funding overview

Funding rounds1 record

Investors

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

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

Frequently asked questions about Skylark Bio