Pipeline ProQR pipeline

ProQR employee working at the lab

ProQR is pioneering a next-generation RNA technology called Axiomer™, which uses a cell’s own editing machinery called ADAR to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for both rare and prevalent diseases with unmet need. Our initial discovery programs aim to explore the potential of our platform across diseases that originate in the liver and nervous system.

ProQR programs

Our pipeline programs share several key characteristics including a deep rooting in human genetics, the potential to have a major impact in a high unmet medical need, the ability to leverage the existing proven delivery technology for delivery to hepatocytes in liver, the opportunity to monitor early biomarkers to establish target engagement in Phase I trials for human proof of concept and availability of well-defined clinical endpoints.

In December 2024, the Rett Syndrome Research Trust and ProQR expaded their RNA editing partnership in Rett Syndrome. Axiomer™ has the potential to restore the precise level of MECP2 protein regulatory function, which is lacking in Rett Syndrome, and become a disease modifying therapy. ​

Click the '+' icon for more information, including estimated population.

AX-0810

Cholestatic Diseases targeting NTCP

Estimated population: ~100.0001

Cholestatic disorders are caused by a buildup of bile acids in the liver. Without treatment, the damage progresses through various stages to ultimately liver failure. Liver transplants are often necessary for primary sclerosing cholangitis (PSC) and biliary atresia (BA), two forms of cholestatic disease where currently there are no approved drugs. AX-0810 is designed to introduce a loss of function (LOF) variant that has been found in human genetics to prevent reuptake of bile acid in liver. Based on its mechanism of action, AX-0810 has the potential to become a disease modifying treatment for a range of cholestatic diseases.

1. Approximately 100.000 people affected with Primary Sclerosing Cholangitis and Biliary atresia in US and EU5.

AX-2402

Rett Syndrome targeting MECP2 R270X

Estimated population: ~20000

Rett syndrome is a progressive neurodevelopmental disorder caused by genetic mutations in the Methyl CpG binding protein 2 (MECP2) and diagnosed primarily in females. It is characterized by apparently normal psychomotor development during the first six to 18 months after birth, followed by a period of developmental stagnation, then a regression in language and motor skills, followed by long-term relative stability. During the phase of regression, affected patients develop repetitive, stereotypic hand movements that replace purposeful hand use. Additional symptoms include gait ataxia and apraxia, seizures, tremors, episodic apnea and/or hyperpnea, gastrointestinal issues, scoliosis and musculoskeletal problems, anxiety and sleep issues and bruxism. 

AX-1412

Cardiovascular Disease targeting B4GALT1

Estimated population: ~200 million2

Cardiovascular diseases (CVDs) are a group of health conditions that affect the heart and blood vessels, such as atherosclerosis which can lead to severe problems like heart attacks, heart failure, and stroke. AX-1412 introduces a variant into B4GALT1 that is associated in human genetics with a significantly lower chance of developing cardiovascular disease. ProQR intends to advance AX-1412 targeting B4GALT1 to early clinical proof of concept stage, then would seek to partner this program.

2. Approximately 200 million people suffer from too high a level of cholesterol in US and EU5. Boonstra K, Beuers U, Ponsioen CY. J Hepatol. 2012 May;56(5):1181-1188; Karlsen TH, et al. J Hepatol. 2017 Dec;67(6):1298-1323; Dyson JK, et al. Lancet. 2018 Jun 23;391(10139):2547-2559; Sundaram SS, et al. Liver Transpl. 2017 Jan;23(1):96-109. Raghu VK, et al. Liver Transpl. 2021 May;27(5):711- 718; NORD, 2019. Tsao CW, et al. Circulation. 2022;145(8):e153–e639. World Health Organization

AX-2911

MASH targeting PNPLA3

Estimated population: ~16 million

AX-1005

Cardiovascular Diseases

Estimated population: ~200 million2

2. Approximately 200 million people suffer from too high a level of cholesterol in US and EU5. Boonstra K, Beuers U, Ponsioen CY. J Hepatol. 2012 May;56(5):1181-1188; Karlsen TH, et al. J Hepatol. 2017 Dec;67(6):1298-1323; Dyson JK, et al. Lancet. 2018 Jun 23;391(10139):2547-2559; Sundaram SS, et al. Liver Transpl. 2017 Jan;23(1):96-109. Raghu VK, et al. Liver Transpl. 2021 May;27(5):711- 718; NORD, 2019. Tsao CW, et al. Circulation. 2022;145(8):e153–e639. World Health Organization

AX-0601

Metabolic Diseases targeting obesity and T2D

Estimated population: ~650 million

AX-9115

Rare Metabolic Condition

Estimated population: ~20.000

AX-2403

Rett Syndrome targeting MECP2 R168X

Estimated population: ~6K

Rett syndrome is a progressive neurodevelopmental disorder caused by genetic mutations in the Methyl CpG binding protein 2 (MECP2) and diagnosed primarily in females. It is characterized by apparently normal psychomotor development during the first six to 18 months after birth, followed by a period of developmental stagnation, then a regression in language and motor skills, followed by long-term relative stability. During the phase of regression, affected patients develop repetitive, stereotypic hand movements that replace purposeful hand use. Additional symptoms include gait ataxia and apraxia, seizures, tremors, episodic apnea and/or hyperpnea, gastrointestinal issues, scoliosis and musculoskeletal problems, anxiety and sleep issues and bruxism. 

AX-2404

Rett Syndrome targeting MECP2 R255X

Estimated population: ~5K

Rett syndrome is a progressive neurodevelopmental disorder caused by genetic mutations in the Methyl CpG binding protein 2 (MECP2) and diagnosed primarily in females. It is characterized by apparently normal psychomotor development during the first six to 18 months after birth, followed by a period of developmental stagnation, then a regression in language and motor skills, followed by long-term relative stability. During the phase of regression, affected patients develop repetitive, stereotypic hand movements that replace purposeful hand use. Additional symptoms include gait ataxia and apraxia, seizures, tremors, episodic apnea and/or hyperpnea, gastrointestinal issues, scoliosis and musculoskeletal problems, anxiety and sleep issues and bruxism. 

AX-2405

Rett Syndrome targeting MECP2 R294X

Estimated population: ~6K

Rett syndrome is a progressive neurodevelopmental disorder caused by genetic mutations in the Methyl CpG binding protein 2 (MECP2) and diagnosed primarily in females. It is characterized by apparently normal psychomotor development during the first six to 18 months after birth, followed by a period of developmental stagnation, then a regression in language and motor skills, followed by long-term relative stability. During the phase of regression, affected patients develop repetitive, stereotypic hand movements that replace purposeful hand use. Additional symptoms include gait ataxia and apraxia, seizures, tremors, episodic apnea and/or hyperpnea, gastrointestinal issues, scoliosis and musculoskeletal problems, anxiety and sleep issues and bruxism. 

AX-2406

Rett Syndrome targeting MECP2 R133H

Rett syndrome is a progressive neurodevelopmental disorder caused by genetic mutations in the Methyl CpG binding protein 2 (MECP2) and diagnosed primarily in females. It is characterized by apparently normal psychomotor development during the first six to 18 months after birth, followed by a period of developmental stagnation, then a regression in language and motor skills, followed by long-term relative stability. During the phase of regression, affected patients develop repetitive, stereotypic hand movements that replace purposeful hand use. Additional symptoms include gait ataxia and apraxia, seizures, tremors, episodic apnea and/or hyperpnea, gastrointestinal issues, scoliosis and musculoskeletal problems, anxiety and sleep issues and bruxism. 

AX-3875

Rare metabolic & CNS disorder

AX-4070

Rare CNS disorder

Partnered pipeline

Eli Lilly and Company

Since 2021 ProQR has had a global licensing and research collaboration with Eli Lilly and Company. The partnership focuses on the discovery, development, and commercialization of potential new medicines for genetic disorders. 

The ongoing collaboration uses ProQR’s proprietary Axiomer™ RNA editing platform to progress multiple RNA editing drug targets toward clinical development and commercialization.

Lilly logo

10 targets (option to expand to 15)

Undisclosed
Partnered with Lilly

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  • Axiomer technology

    At ProQR we invented the Axiomer® technology that enables single base editing to target currently untreatable diseases. Find out more on our dedicated webpages.

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  • Working at ProQR - Careers

    We are a biotechnology company focused on the development of life changing RNA therapies for rare and common diseases. Want to learn more? Visit our dedicated careers page.

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  • Scientific presentations and publications

    Find our groundbreaking science underlying our pipeline programs in our scientific presentations and publications webpage.

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