What GondolaBio does

GondolaBio, LLC is a clinical-stage biopharmaceutical company founded in September 2024 as a spinout of BridgeBio Pharma, operating as a sister company under a decentralized subsidiary model. The company is headquartered in Palo Alto, California, and is led by Co-founder and CEO Neil Kumar, with Morgan Paull as COO, James Li as President, and Pete Schmidt as Chief Medical Officer of its affiliate Portal Therapeutics. GondolaBio is focused on developing disease-modifying therapeutics for patients with rare genetic diseases, with a stated emphasis on indications lacking approved treatments.

The company's lead asset is PORT-77, an oral small molecule inhibitor of the ABCG2 export protein designed to reduce plasma protoporphyrin IX (PPIX) concentrations in erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP). Phase 2a GATEWAY results reported 79% plasma PPIX reduction in the high-dose cohort and 63% in the low-dose cohort with no serious adverse events, and the program has received FDA Orphan Drug and Fast Track Designations. Beyond PORT-77, the pipeline spans approximately 15 programs across indications including autosomal dominant polycystic kidney disease, alpha-1 antitrypsin deficiency, Charcot-Marie-Tooth 1A, neurofibromatosis type 1, hereditary pancreatitis, tuberous sclerosis complex, genetic epilepsies, and others, with an additional antisense oligonucleotide (ASO) discovery capability added via a partnership with the n-Lorem Foundation.

The business is pre-revenue and operates under a capital-intensive clinical development model, backed by a $300 million founding financing from Patient Square Capital, Viking Global Investors, and Frazier Life Sciences, with additional participation from Aisling Capital, Cormorant Asset Management, and Sequoia Capital. GondolaBio is organized into multiple wholly-owned subsidiaries (8 formed within the first 3 months of operation), each housing a discrete drug development program. Future commercialization is expected to follow a specialty pharma / orphan drug pricing model, with distribution through specialty pharmacies and rare disease specialists, and the company has already stood up an Expanded Access Program as pre-launch infrastructure.

Name

GondolaBio

Legal name

GondolaBio, LLC

Website

https://gondolabio.com

Company type

Private

Founded year

2024

Operating status

Operating

Headcount range

101–250 employees

Short description

GondolaBio is a clinical-stage biopharmaceutical company developing disease-modifying therapeutics for rare genetic diseases, led by oral ABCG2 inhibitor PORT-77 for EPP and XLP, and operating a decentralized subsidiary model with 15+ pipeline programs as a spinout of BridgeBio Pharma.

Ownership category

akta.pro rank

Product category

Rare Disease Biopharmaceuticals

NAICS

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

SIC

Services-Commercial Physical & Biological Research (8731), Pharmaceutical Preparations (2834)

akta.pro primary industry

Antisense Oligonucleotides (ASO) Therapeutics (splice modulation/knockdown) (HLAAADAC)

akta.pro secondary industry

RNA Therapeutics Regulatory, Quality & Pharmacovigilance (GxP, comparability, post-market) (HLAAADAJ)

Keywords

• Rare disease therapeutics
• Genetic disease medicines
• Small molecule inhibitors
• Clinical-stage biopharmaceuticals
• Orphan drug development

Headquarters

HQ city

Palo Alto

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. Pharmaceutical Product Sales: Pre-revenue clinical stage company. Upon regulatory approval, the company plans to commercialize PORT-77 and other pipeline candidates for genetic diseases. Revenue model will likely follow specialty pharma pricing with orphan drug pricing premiums.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels5 records

Core offering

GondolaBio is a clinical-stage biopharmaceutical company developing oral small molecule and antisense oligonucleotide (ASO) therapeutics for rare genetic diseases. The lead program, PORT-77, is an oral ABCG2 inhibitor in Phase 2 development for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP), with a broader pipeline of 15+ programs spanning autosomal dominant polycystic kidney disease, alpha-1 antitrypsin deficiency, tuberous sclerosis complex, neurofibromatosis type 1, Charcot-Marie-Tooth 1A, and other genetic conditions. The company operates through a decentralized model with eight subsidiary companies, each housing an individual drug development program.

Product overview

GondolaBio is a clinical-stage biopharmaceutical company developing breakthrough therapeutics for genetic diseases. The company's portfolio centers on PORT-77 as its lead program—an oral ABCG2 inhibitor for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP)—currently in Phase 2 development with Phase 2a results demonstrating 79% plasma PPIX reduction. The broader pipeline includes multiple genetic disease programs spanning discovery through IND-enabling stages, with a decentralized subsidiary model enabling parallel development of distinct therapeutic programs.

Differentiator

Problem solved

Functional benefit

Products and services

• PORT-77 PORT-77 is an oral, small molecule ABCG2 inhibitor in Phase 2 development for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP). It is designed to reduce plasma protoporphyrin IX (PPIX) concentrations by inhibiting ABCG2-mediated efflux from red blood cells and hepatocytes, with the goal of addressing skin phototoxicity and liver damage in EPP and XLP patients.
• Genetic Disease Pipeline A diverse pipeline of 15+ drug development programs across multiple rare genetic diseases including autosomal dominant polycystic kidney disease, alpha-1 antitrypsin deficiency, Charcot-Marie-Tooth 1A, neurofibromatosis type 1, hereditary pancreatitis, tuberous sclerosis complex 1/2, genetic epilepsy driven by SynGAP1 mutations, Dup15q developmental epileptic encephalopathy, recurrent oxalate kidney stones, fibrous dysplasia, best vitelliform macular dystrophy, early onset preeclampsia, and 4 undisclosed programs.

Quantifiable outcome

• 79% plasma PPIX reduction in high-dose cohort (300mg BID)
• +3 more outcomes

Segments1 record

Ideal customer profiles1 record

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Five partnerships are on record, tiered minor and core.

• American Porphyrias Expert Collaborative (APEX)minorStrategic or Co-development Partner · 22 June 2026Partnership for clinician webinar on PORT-77 Phase 2a GATEWAY study clinical data and disease-modifying potential in EPP.
• International Porphyria Network (IPNET)minorStrategic or Co-development Partner · 22 June 2026Partnership for clinician webinar on PORT-77 clinical data.
• Global Porphyria Advocacy Coalition (GPAC)minorStrategic or Co-development Partner · 22 June 2026Partnership for patient webinar to help the EPP community understand what the Phase 2a GATEWAY study means for them.
• n-Lorem FoundationcoreStrategic or Co-development Partner · 17 December 2024Multi-collaboration agreement to discover novel antisense oligonucleotide (ASO) medicines for two prespecified biological targets in genetic diseases with significant unmet need. n-Lorem provides AI-informed ASO discovery platform and expertise; GondolaBio funds research and advances resulting therapies through clinical and commercial development via subsidiary companies. This partnership added the seventh and eighth subsidiaries to GondolaBio's portfolio.
• BridgeBio PharmacoreStrategic or Co-development Partner · 1 September 2024GondolaBio was spun out from BridgeBio Pharma in September 2024 as a sister company, with three early-stage genetic disease assets transferred for development. GondolaBio leverages BridgeBio's decentralized model and drug development expertise.

Scale indicators4 records

Recent moves7 records

Expansion highlights8 records

Direct peers

• BridgeBio Pharma: Sister company and GondolaBio's parent at spinout; BridgeBio operates the same decentralized genetic-disease model and has commercialized rare-disease assets (e.g., Attruby for ATTR-CM). Direct operational and strategic peer given shared leadership lineage, capital structure philosophy, and target disease areas.
• Ionis Pharmaceuticals: Leading antisense oligonucleotide (ASO) therapeutics company with multiple approved rare-disease drugs (Spinraza, Tegsedi, Wainua). Closely comparable to GondolaBio's ASO pipeline ambitions via its n-Lorem partnership given identical nucleic-acid modality and rare-disease focus.
• Alnylam Pharmaceuticals: RNA-targeted therapeutics leader with multiple approved rare-disease products (Onpattro, Givlaari, Oxlumo, Amvuttra). Comparable in modality (RNA therapeutics), commercial-stage rare-disease execution, and pipeline breadth targeting genetic diseases with high unmet need.
• Ultragenyx Pharmaceutical: Clinical and commercial-stage rare-disease biotech with multiple approved products (Crysvita, Dojolvi, Mepsevii) and a pipeline targeting genetic conditions. Highly comparable decentralized development approach, orphan-disease focus, and similar small-molecule and biologic modality mix.
• Regenxbio: Clinical-stage gene therapy biotech targeting rare genetic diseases (ABCC6, Hunter syndrome, Duchenne) with platform technology. Comparable in genetic-disease focus, clinical-stage profile, and orphan-drug regulatory strategy, though different modality (AAV gene therapy vs. small molecule/ASO).

Broad incumbents

• BioMarin Pharmaceutical: Established rare-disease biopharma with multiple approved genetic-disease products (Vimizim, Naglazyme, Voxzogo, Roctavian). Directly comparable target market (rare genetic diseases), specialty pharma commercialization model, and focus on disease-modifying treatments for underserved populations.
• Sarepta Therapeutics: Genetic-disease specialty biopharma with approved treatments for Duchenne muscular dystrophy and expanding gene therapy franchise. Comparable in genetic-disease focus, specialty distribution model, and orphan-drug commercial strategy, though focused on neuromuscular conditions.
• Vertex Pharmaceuticals: Large-cap biopharma with genetic-disease franchises in cystic fibrosis (Trikafta) and emerging programs in sickle cell disease, alpha-1 antitrypsin deficiency, and APOL1-mediated kidney disease — directly overlapping GondolaBio's A1AT and ADPKD programs.

Emerging players

• Solid Biosciences: Clinical-stage genetic-disease biotech developing treatments for Duchenne muscular dystrophy and other rare conditions. Comparable in clinical-stage profile, rare genetic-disease focus, and small-cap valuation trajectory, though narrower therapeutic area focus.
• Entrada Therapeutics: Clinical-stage biotech developing intracellular enzyme therapeutics for genetic diseases including Duchenne and myotonic dystrophy. Comparable in clinical-stage rare-disease positioning, platform approach to genetic diseases, and capital scale similar to GondolaBio's pre-IPO profile.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks6 records

Key highlights7 records

Customer concentration

Revenue estimate

Valuation estimate

Number of profiles

Profiles4 records

Subsidiaries3 records

Funding overview

Funding rounds1 record

Investors6 records

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 GondolaBio