Purespring Therapeutics

What Purespring Therapeutics does

Purespring Therapeutics is a clinical-stage precision nephrology company developing gene therapies for glomerular kidney diseases. Founded in July 2021 by Syncona Limited with £45m Series A funding and based in London, the company is built around the proprietary GlomThera™ platform — an AAV gene therapy delivery system that targets podocytes, the specialised non-dividing cells implicated in approximately 60% of renal diseases. By using direct kidney delivery via routine interventional radiology procedures, GlomThera achieves 89% ± 11% glomerular transduction in large animal studies while requiring dosing more than 10-fold lower than systemically administered AAV gene therapies, thereby minimising systemic exposure. The lead candidate, PS-002, is an AAV-based therapy delivering complement factor I to podocytes for treatment of IgA nephropathy (IgAN) — the most common primary glomerulonephritis worldwide — and entered Phase I/II clinical trials in 2025 following FDA IND clearance (July 2025), UK MHRA CTA approval (August 2025), and EMA Orphan Drug Designation (April 2025). The pipeline also includes PS-001 for NPHS2-mediated focal segmental glomerulosclerosis (FSGS) and PS-003 for Alport Syndrome, while a secondary proprietary asset, the FunSel in-vivo screening platform (developed by co-founder Professor Mauro Giacca), has been licensed to SwanBio Therapeutics for neurological applications generating milestone and royalty rights.

Purespring is pre-revenue and has raised £115m ($150m) total across two funding rounds: the £45m Series A (July 2021) and an oversubscribed £80m/$105m Series B (October 2024) led by Sofinnova Partners with Gilde Healthcare, Forbion, British Patient Capital, and founding investor Syncona. The company's commercial model is dual: future one-time licensing/product revenue from approved gene therapies distributed through specialty pharmacy and hospital channels, plus platform licensing royalties from the FunSel partnership. The GTM approach is enterprise-focused on regulatory advancement and KOL engagement, with active participation at ASN Kidney Week, ERA Congress, and investor conferences including Piper Sandler and Chardan.

Name

Purespring Therapeutics

Legal name

Purespring Therapeutics Limited

Website

https://purespringtx.com

Company type

Private

Founded year

2021

Operating status

Operating

Headcount range

11–50 employees

Short description

Purespring Therapeutics is a clinical-stage gene therapy company developing AAV-based treatments delivered directly to kidney podocytes via its proprietary GlomThera™ platform, with lead candidate PS-002 in Phase I/II trials for IgA nephropathy.

Ownership category

akta.pro rank

Product category

Gene Therapy / Precision Nephrology

NAICS

Medical and Diagnostic Laboratories (6215)

SIC

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

akta.pro industry

PRIMARYHLAIAIAI

Rare Renal & Nephrology Disorder Therapies

Healthcare & Life Sciences/Pharmaceuticals/Orphan & Rare Disease Pharmaceuticals

SECONDARYHLAKAHAG

Glomerular & Autoimmune Kidney Disease (Glomerulonephritis) Clinics

Healthcare & Life Sciences/Specialist Medical Practice/Nephrology

SECONDARYHLAKAHAO

Genetic & Rare Kidney Disease Clinics

Healthcare & Life Sciences/Specialist Medical Practice/Nephrology

SECONDARYHLAHAKAK

Renal Care Ancillaries (Anemia Management Devices, Phosphate Binder Support, Nutrition Monitoring)

Healthcare & Life Sciences/Medical Devices & Diagnostics/Renal & Dialysis Devices

Keywords

• AAV gene therapy
• Kidney disease treatments
• Glomerular disease therapies
• Precision nephrology platform
• Podocyte-targeted therapeutics

Headquarters

HQ city

London

HQ country

United Kingdom

HQ region

Europe

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Personnel

Revenue model

1. Gene Therapy Product Revenue: Pre-revenue clinical-stage biotech company developing genetic therapies for kidney diseases. Expected to generate revenue from commercialised gene therapy products following regulatory approval. Pipeline includes PS-002 for IgA nephropathy, PS-001 for NPHS2-mediated FSGS, and PS-003 for Alport Syndrome.
2. Platform Licensing and Partnerships: The FunSel screening platform has been licensed to SwanBio Therapeutics for neurological applications, generating potential milestone payments and royalties upon commercialisation. Additional platform partnering opportunities being explored.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels8 records

Core offering

Purespring Therapeutics is a clinical-stage precision nephrology company developing AAV-based gene therapies to treat glomerular kidney diseases. Its proprietary GlomThera™ platform delivers genetic payloads directly to podocytes in the kidney, and its lead candidate PS-002 (complement factor I gene therapy) is in Phase I/II clinical trials for IgA nephropathy, with additional programs PS-001 (NPHS2-mediated FSGS) and PS-003 (Alport Syndrome) in preclinical development.

Product overview

Purespring Therapeutics is a precision nephrology company with a platform-plus-products architecture. The company's core proprietary GlomThera™ platform enables targeted delivery of genetic therapies directly to podocytes using AAV vectors. Lead product PS-002 delivers complement factor I to treat IgA nephropathy, currently in Phase I/II clinical trials. PS-001 targets NPHS2-mediated FSGS and PS-003 targets Alport Syndrome. The FunSel screening platform supports target discovery. The pipeline addresses both rare monogenic disorders and complement-driven glomerular diseases with the goal of providing disease-modifying, potentially curative treatments.

Differentiator

Problem solved

Functional benefit

Products and services

• GlomThera™ Platform Proprietary AAV gene therapy platform that delivers genetic therapies directly to podocytes in the kidney, using a validated AAV capsid for precision targeting with dosing more than 10-fold lower than systemically administered AAV gene therapies. Licensed to third parties for non-renal applications via the FunSel platform (e.g., SwanBio Therapeutics).
• PS-002 (Complement Factor I Gene Therapy) Lead AAV-based gene therapy delivering complement factor I (CFI) gene to podocytes for the treatment of IgA nephropathy (IgAN), targeting the complement pathway known to drive the disease without affecting systemic complement activation. Designed for single-administration, durable efficacy.
• PS-001 (NPHS2 Gene Therapy) Gene therapy candidate in development for treatment of NPHS2-mediated focal segmental glomerulosclerosis (FSGS), designed to replace defective NPHS2 genes encoding podocin.
• PS-003 (Alport Syndrome Gene Therapy Programme) Gene therapy programme targeting Alport Syndrome, a genetic kidney disease with significant unmet need and limited treatment options for patients.
• FunSel Screening Platform Proprietary in-vivo 'gene search engine' that enables functional selection of therapeutic factors unconstrained by previous drug discovery bias, using a library of thousands of AAV vectors encoding secreted proteins. Licensed to third-party biotechs for target discovery outside renal indications.

Quantifiable outcome

• 89% ± 11% kidney glomeruli transduction achieved in large animal studies
• +3 more outcomes

Segments3 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature5 records

Partnerships

Six partnerships are on record, tiered core and minor.

• Bristol Renal at University of BristolcoreStrategic or Co-development Partner · 29 April 2026Collaboration on population modelling study published in Kidney International Reports analysing NPHS2 variants as cause of adult-onset FSGS. Study involved analysis of over 1.2 million genomes from five international databases. Purespring co-founder Professor Moin Saleem leads Bristol Renal research group.
• NateraminorStrategic or Co-development Partner · 29 April 2026Collaboration partner in NPHS2 research study. Natera's RenaSight™ platform used for real-world cohort analysis validating population modelling data showing pathogenic NPHS2 variants as significant cause of adult-onset FSGS.
• Travere TherapeuticsminorStrategic or Co-development Partner · 29 April 2026Collaboration partner in NPHS2 research study. Clinical cohort data from Travere's DUPLEX study supported findings on NPHS2-associated disease prevalence and variant patterns.
• GescapeminorStrategic or Co-development Partner · 29 April 2026Collaboration partner in NPHS2 research study contributing to population modelling of genetic variants across international databases.
• Chromatin BiosciencecoreTechnology or Integration · 27 October 2025Research collaboration to develop cell-specific gene therapy promoters leveraging Chromatin's proprietary platform. Collaboration aims to improve gene delivery safety and efficacy, supporting Purespring's pipeline targeting kidney diseases including IgA nephropathy.
• SwanBio TherapeuticscoreTechnology or Integration · 18 July 2022Licensing agreement for use of FunSel screening platform. SwanBio licensed FunSel to explore new targets for early-stage pipeline programs in neurological diseases. SwanBio has option to develop identified targets and own/commercialise resulting molecules. Purespring entitled to milestones and royalties upon successful commercialisation.

Scale indicators4 records

Recent moves7 records

Expansion highlights5 records

Direct peers

• Travere Therapeutics: Direct competitor in IgA nephropathy with FDA-approved Filspari (sparsentan) and active FSGS programs. Partnership data contributor on Purespring's NPHS2 population study. Most directly comparable commercial-stage peer.
• Calliditas Therapeutics (Asahi Kasei): Developer of Tarpeyo (delayed-release budesonide), the first FDA-approved treatment specifically for IgA nephropathy. Direct competitor for Purespring's lead PS-002 indication with established commercial infrastructure.
• Chinook Therapeutics (Novartis): Kidney disease-focused biotech (acquired by Novartis in 2023) developing treatments for IgAN and other rare kidney diseases. Closely comparable business model and indication focus to Purespring.

Emerging players

• Apellis Pharmaceuticals: Complement pathway-focused biotech with Empaveli/Aspaveli (pegcetacoplan) approved for PNA and in development for kidney indications including IgAN. Mechanistically relevant peer given PS-002 targets complement pathway.
• Arrowhead Pharmaceuticals: RNAi-based therapeutics developer with active kidney disease programs including IgAN. Comparable in being a development-stage biotech targeting glomerular diseases with novel modality approaches.
• uniQure: Clinical-stage AAV gene therapy company with approved Hemgenix (with CSL) for hemophilia B. Direct gene therapy platform peer navigating similar regulatory, manufacturing, and commercialization challenges.
• Sangamo Therapeutics: Clinical-stage gene therapy and genome editing company with programs across multiple therapeutic areas. Comparable as an AAV-based platform biotech facing similar manufacturing and clinical execution challenges.

Broad incumbents

• BioMarin Pharmaceutical: Established commercial-stage gene therapy company with multiple approved products (Roctavian, Hemgenix). Comparable in gene therapy modality and rare disease focus, though with broader pipeline.
• Vertex Pharmaceuticals: Major rare disease biopharma with growing gene therapy portfolio through Alpine Immune Sciences and other acquisitions. Established peer in rare genetic disease space and a potential acquirer profile.
• BridgeBio Pharma: Genetic disease-focused biopharma with multiple programs including in rare renal conditions. Relevant comparable for genetic disease drug development model with broad pipeline and orphan drug strategy.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks5 records

Key highlights6 records

Customer concentration

• Xx.com/PURESPRINGTX
• LinkedInlinkedin.com/company/purespring-therapeutics

Revenue estimate

Valuation estimate

Number of profiles

Profiles16 records

Funding overview

Funding rounds2 records

Investors5 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 Purespring Therapeutics