Creating New and Meaningful Medicines for Patients with Genetic Diseases
We are a clinical-stage biopharmaceutical company developing breakthrough therapeutics for patients living with genetic diseases, focused on translating research and development advances into transformative medicines for patients.
A sister company of BridgeBio Pharma, GondolaBio launched in 2024 with a lean, decentralized approach to drug development with a focus on early-stage research and development for genetic diseases.
With a mission centered around helping as many patients as quickly as possible, we are focused on identifying clear genetic markers to target these conditions at their source. Coupled with our unique, decentralized business model, we are well-positioned to accelerate opportunities for patients who are often underserved by traditional pharma models.
Daniel, living with TSC
An Unwavering Commitment to Underserved Patients
Nearly 30 million people in the U.S. are living with genetic diseases, and half are children. Despite this prevalence and significant strides toward understanding the underlying causes of many genetic diseases, only 5% of these conditions have approved therapies. People living with rare diseases often fare the worst, with companies unable to dedicate the resources needed to find new and effective treatments.
At GondolaBio, we are looking to address this gap through rigorous and cutting-edge science. By focusing our efforts where the need is greatest and the science is strongest, we are centering the patient experience in every decision that we make, with a goal of improving the lives of these tens of millions of individuals who are in need of new treatments.
Our Pipeline
We’re building a diverse pipeline in genetic disease, spanning a variety of diseases and modalities.
| Indication | Patient Population (US+EU) |
Discovery
Lead Op
IND Enabling
Phase 1
Phase 2
|
|---|---|---|
| Erythropoietic Protoporphyria | 25k | |
| Alpha-1 Antitrypsin Deficiency | 200k | |
| Hereditary Pancreatitis | 30k | |
| Neurofibromatosis Type 1 | 200k | |
| Fibrous Dysplasia | 50k | |
| Autosomal Dominant Polycystic Kidney Disease | 300k | |
| Recurrent Oxalate Kidney Stones | 300k | |
| Tuberous Sclerosis Complex 1/2 | 65k | |
| Genetic Epilepsy Driven by SynGAP1 Mutations | 15k | |
| Dup15q Developmental Epileptic Encephalopathy | 20k | |
| Angelman Syndrome | 50k | |
| +7 Undisclosed Programs |
PORT-77, a potential breakthrough treatment for people living with EPP and XLP
EPP and XLP are rare genetic conditions that have significant morbidity and impact on quality of life
Through our lead program, we are seeking to develop a best-in-class disease-modifying treatment for erythropoeitic protoporphyria (EPP) and X-linked protoporphyria (XLP).
EPP and XLP are rare genetic conditions affecting greater than 25,000 people in the U.S. and Europe, in which the body produces too much of a compound called protoporphyrin IX (PPIX) that can accumulate in the liver and skin. This leads to extreme sensitivity to sunlight, causing painful skin reactions including burning and swelling, and in approximately 20-30% of individuals, liver damage, with up to 5% experiencing liver failure requiring transplant.
Treatment options for people living with EPP or XLP are extremely limited, with no approved disease-modifying treatments. Individuals are primarily instructed to avoid sunlight to manage symptoms, a precaution that severely limits their quality of life.
PORT-77: A Potentially Best-in-Class Disease-Modifying Treatment for EPP and XLP
Our lead investigational program, PORT-77, is an oral, small molecule ABCG2 inhibitor currently in Phase 2 development. PORT-77 has the potential to become a best-in-class treatment for EPP and XLP.
The ABCG2 export protein mediates the transport of PPIX from red blood cells and precursors into the plasma. We hypothesize that inhibition of ABCG2 by PORT-77 decreases PPIX efflux and reduces plasma PPIX concentrations, potentially ameliorating PPIX-mediated skin phototoxicity and liver damage.
We envision a brighter future for people living with EPP and XLP, in which PORT-77 alleviates their symptoms, thereby improving quality of life.
>25,000
People affected by EPP in the US & Europe
PORT-77 is an investigational product whose efficacy and safety have not been evaluated by any Health Authority.
Morgan, living with Erythropoietic Protoporphyria (EPP)
Join our team and help us shape the future of biopharma
Reengineering the Future of Biopharma
Interested in a role that enables you to define white space, push boundaries and help solve life-changing problems for patients? If you are someone who defies convention, then consider joining our team of experienced drug discoverers, developers and innovators committed to translating advances in genetic science and medicine.
Together, we’ll challenge the status quo, asking “why not?” at every turn, to consistently do right by patients and help reengineer the future of biopharma.
Open Positions
View All Open Positions
MORGAN, Age 14, Living with EPP
Expanded Access Policy
GondolaBio is committed to evaluating all requests in a fair and equitable manner. All requests must be submitted by the patient’s treating physician; GondolaBio may require more detailed information to fully evaluate a request. Each request will be given careful consideration by GondolaBio whose decisions are final. Physicians seeking pre-approval access for patients with no alternative treatment options should submit their requests to medinfo@gondolabio.com.