What Spirovant does

Spirovant Sciences is a clinical-stage gene therapy company developing inhaled AAV-based treatments for cystic fibrosis and other genetic respiratory diseases. The company was founded in 2016 as Talee Bio and is now headquartered in Philadelphia, PA, at uCity Square (3675 Market Street, Suite 900). Its core technology is the proprietary AAV + Augmenter platform, which combines a novel AAV capsid (AAV2.5T) engineered for high tropism to the apical surface of human airway epithelia, an hCFTRΔR minigene payload, and doxorubicin as a small-molecule transduction augmenter that inhibits proteasome activity to enhance nuclear translocation of the AAV genome. The lead candidate SP-101 is delivered via aerosolized inhalation and is being evaluated in the SAAVe Phase 1/2 multicenter trial (first patient dosed November 2024) for CF patients who do not benefit from CFTR modulators — approximately 10–20% of the estimated 82,000 CF patients worldwide, including those with Class I nonsense mutations. SP-101 and its predecessor SPIRO-2101 have received FDA Orphan Drug Designation and Rare Pediatric Disease Designation.

The company operates as a wholly-owned subsidiary of Sumitovant Biopharma Ltd. (itself a subsidiary of Sumitomo Dainippon Pharma), though in 2024 Chief Scientific Officer Roland Kolbeck led a management buy-out making him co-owner. Spirovant is pre-revenue; its funding to date is modest (a 2017 SBIR grant of $225,000 and a $4.5 million Cystic Fibrosis Foundation award in 2019), with the balance of capital historically provided through the parent company. The go-to-market is clinical and partnership-based: multicenter academic trial sites (e.g., Columbia University Irving Medical Center), with future commercialization expected through specialty pharmacy channels typical of rare-disease gene therapies. Scientific advisors include senior CF gene therapy researchers from the University of Iowa and the Children's Hospital of Philadelphia, and the company holds Cystic Fibrosis Foundation scientific endorsement.

Name

Spirovant

Legal name

Spirovant Sciences Inc.

Website

https://spirovant.com

Company type

Private

Founded year

2016

Operating status

Operating

Headcount range

11–50 employees

Short description

Spirovant Sciences is a clinical-stage gene therapy company developing inhaled AAV-based treatments (lead candidate SP-101) for cystic fibrosis patients who do not benefit from CFTR modulators, using a proprietary AAV + Augmenter platform that combines a novel AAV2.5T capsid with doxorubicin as a transduction enhancer.

Ownership category

akta.pro rank

Product category

Gene Therapy for Respiratory Diseases

NAICS

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

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Cystic Fibrosis & Bronchiectasis Care (HLAKANAK)

akta.pro secondary industry

Pulmonary Hypertension & Rare Pulmonary Disease Therapies (HLAIAIAG)

Keywords

• Gene therapy development
• Cystic fibrosis treatment
• AAV vector platforms
• Respiratory disease therapeutics
• Clinical stage biotechnology

Headquarters

HQ city

Pausa

HQ country

Germany

HQ region

Europe

Offices1 record

Markets served

GTM type

B2B

Offering type

Services

Cost components

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

Revenue model

1. Gene Therapy Development and Commercialization: As a clinical-stage biotech company, Spirovant's revenue model is centered on developing and commercializing gene therapy candidates for cystic fibrosis and other respiratory diseases. The company is advancing SP-101 through clinical trials with the goal of regulatory approval and commercialization. As a wholly-owned subsidiary of Sumitovant Biopharma, funding comes through parent company investment rather than traditional product revenue at this stage.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels4 records

Core offering

Spirovant Sciences is a clinical-stage gene therapy company developing inhaled AAV-based treatments for cystic fibrosis and other respiratory diseases. The company's lead candidate, SP-101, uses a proprietary AAV + Augmenter platform combining a novel AAV2.5T capsid with doxorubicin as a transduction augmenter to deliver functional CFTR protein to airway epithelia. SP-101 is currently in Phase 1/2 SAAVe clinical trials (first patient dosed November 2024) for CF patients who do not benefit from CFTR modulator therapies.

Product overview

Spirovant Sciences is a gene therapy company developing treatments for genetic lung diseases, with a focus on cystic fibrosis (CF). The company operates primarily through its proprietary AAV + Augmenter Platform, which enables the delivery of functional CFTR genes to airway cells via aerosolized administration. The core product portfolio includes SP-101 (currently in SAAVe Phase 1/2 clinical trials for CF), SPIRO-2101 (in development with FDA Orphan Drug and Rare Pediatric Disease Designations), and an undisclosed respiratory AAV program in preclinical stage. SP-101 uses a novel AAV capsid (AAV2.5T) combined with doxorubicin as a transduction augmenter to achieve high levels of CFTR expression. The company is a clinical-stage gene therapy company and wholly-owned subsidiary of Sumitovant Biopharma.

Differentiator

Problem solved

Functional benefit

Products and services

• SP-101 SP-101 is an investigational recombinant adeno-associated virus (AAV) gene therapy optimized for high tropism to human airway epithelia. It is composed of a novel AAV capsid (AAV2.5T) and a human CFTR minigene (hCFTRΔR), administered via inhalation in combination with doxorubicin as an augmenter to enhance transgene expression. Currently in Phase 1/2 SAAVe clinical trials for cystic fibrosis patients who do not benefit from CFTR modulator therapies. Has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation.
• SPIRO-2101 SPIRO-2101 is an inhaled adeno-associated virus (AAV) gene therapy designed to replace a defective cystic fibrosis transmembrane conductance regulator (CFTR) gene in patients with class 1 mutations or those unable to tolerate existing CFTR modulators. It contains an evolved AAV capsid engineered for high tropism to human airway epithelia and has received FDA Orphan Drug Designation and Rare Pediatric Disease Designations.
• Undisclosed Respiratory AAV Program

Quantifiable outcome

• Dosing the first patient in SAAVe Phase 1/2 clinical trial in November 2024 - a major regulatory milestone for the company and the CF patient community
• +2 more outcomes

Named customers1 record

Segments2 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Four partnerships are on record, tiered core and minor.

• Columbia University Irving Medical CentercoreStrategic or Co-development PartnerDr. Claire Keating, Associate Professor of Medicine and Co-Director of the Gunnar Esiason Adult Cystic Fibrosis and Lung Center at Columbia University Irving Medical Center, is participating in the SAAVe Phase 1/2 clinical trial.
• University of PennsylvaniaminorStrategic or Co-development PartnerCEO Joan Lau serves as an adjunct instructor at the University of Pennsylvania and serves on the Roy & Diana Vagelos Program in Life Sciences Management board. The company is located in Philadelphia's thriving life sciences corridor near leading universities.
• University City Science CenterminorStrategic or Co-development PartnerSpirovant received first investment and ongoing commercialization support from the University City Science Center, located in Philadelphia's uCity Square knowledge community.
• Wexford Science and Technology (uCity Square)minorStrategic or Co-development PartnerSpirovant's headquarters and laboratory are located in uCity Square, developed by Wexford Science and Technology, a knowledge community supporting life sciences companies.

Scale indicators5 records

Recent moves7 records

Expansion highlights5 records

Emerging players

• Krystal Biotech: Krystal Biotech is a commercial-stage gene therapy company (Vyjuvek for dystrophic epidermolysis bullosa) developing inhaled AAV-based therapies for pulmonary diseases including cystic fibrosis and alpha-1 antitrypsin deficiency. Comparable as a small-cap AAV gene therapy company targeting respiratory indications with regulatory designations and platform-based pipeline expansion.
• Trizell (formerly Abeona Therapeutics CF program): Trizell developed ulocuplumab and is pursuing gene therapy for alpha-1 antitrypsin deficiency and other respiratory diseases. Comparable as a clinical-stage gene therapy company developing AAV-based treatments for genetic lung diseases, sharing platform modality and respiratory disease focus with Spirovan.
• Eloxx Pharmaceuticals: Eloxx developed ELX-02, a ribosomal readthrough agent for CF patients with nonsense mutations (Class I). Comparable as a clinical-stage company targeting the CF non-modulator population with a different modality (small molecule readthrough) addressing the same genetic basis Spirovant targets via gene replacement.
• ReCode Therapeutics: ReCode is developing RCT1100, an inhaled mRNA therapy for cystic fibrosis using its selective organ targeting (SORT) lipid nanoparticle platform. Like Spirovant, ReCode targets CF patients not eligible for CFTR modulators and pursues an inhaled, mutation-agnostic approach to address the underlying genetic cause.
• Beam Therapeutics: Beam Therapeutics develops base editing therapies including for cystic fibrosis (BEAM-301 targeting CFTR nonsense mutations). Comparable as a clinical-stage genetic medicines company pursuing mutation-agnostic or mutation-specific approaches to treat CF patients not responsive to CFTR modulators, with deep scientific advisors and regulatory pathway understanding.
• Arcturus Therapeutics: Arcturus develops LUNAR lipid nanoparticle platform for mRNA therapeutics including inhaled lung-targeted applications (ARCT-032 for CF). Comparable as a clinical-stage nucleic acid therapeutics company developing inhaled CF therapies that could compete with Spirovant's AAV approach in the same non-responder patient population.
• ProQR Therapeutics: ProQR develops RNA-editing therapies (Axiomer) for genetic diseases including cystic fibrosis and other respiratory conditions. Comparable as a clinical-stage genetic medicines company targeting CF with mutation-agnostic or mutation-specific approaches to address the unmet need for non-modulator-eligible patients.

Direct peers

• Translate Bio (Sanofi): Translate Bio, acquired by Sanofi, developed MRT5005, an inhaled mRNA therapy for cystic fibrosis. Both Spirovant and Translate Bio pursue inhaled nucleic acid-based therapies for CF lung disease, targeting the same patient population with mutation-agnostic approaches, although Translate Bio uses mRNA rather than AAV.
• 4D Molecular Therapeutics: 4DMT is developing 4D-710, an inhaled AAV gene therapy for cystic fibrosis lung disease using a synthetic aerosolized capsid (A101). Both Spirovant and 4DMT pursue inhaled AAV gene therapy for CF, addressing the same patient population and biological delivery challenges, making them direct competitors in the CF genetic medicine space.

Broad incumbents

• Vertex Pharmaceuticals: Vertex is the dominant CF therapeutics company with Trikafta/Kaftrio and other CFTR modulators treating ~90% of CF patients. As the standard of care, Vertex is the primary competitive benchmark Spirovant must address — its efficacy, safety, and physician familiarity set the bar any gene therapy must beat for non-responders and potentially the broader population.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights6 records

Customer concentration

• Xx.com/spirovant
• LinkedInlinkedin.com/company/spirovant
• Facebookfacebook.com/100942561371444

Compliance4 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles7 records

Funding overview

Funding rounds2 records

Investors2 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 Spirovant