The funding will be used to progress the development of ABX1100 to potentially treat Pompe disease. Credit: Krakenimages.com / Shutterstock.com.

Clinical-stage biotechnology company​ Aro Biotherapeutics has raised $41.5m in a Series B financing round to advance the development of its lead candidate, ABX1100, for Pompe disease.

Cowen Healthcare Investments led the funding round with a syndicate of Series A investors, including Northpond Ventures, Johnson & Johnson Innovation – JJDC, BVF Partners and Healthcap taking part.

Go deeper with GlobalData

Premium Insights

The gold standard of business intelligence.

Find out more

JJDC president Christopher Picariello has joined the board of Aro.

The company recently commenced a first-in-human clinical trial of the therapy with initial findings expected in 2024.

ABX1100 comprises a Centyrin attaching to the CD71 receptor conjugated to a small interfering RNA (siRNA) which hinders GYS1 mRNA expression. 

See Also:

The treatment reduces GYS1 enzyme levels, lowering its activity in muscle tissues. 

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

In good laboratory practice toxicology studies, the therapy showed a favourable safety profile with a lasting decline in GYS1 mRNA in muscles that support dosing every month or quarter. 

The US Food and Drug Administration had previously granted orphan drug and rare paediatric disease designations to ABX1100.

The funds will aid the company in progressing the development of Centyrin-siRNA conjugate programmes to discover new autoimmune disorder treatments. 

​Aro CEO Susan Dillon stated: ​“We are appreciative of the support provided by this syndicate of leading life science industry investors. 

“Aro is well positioned to advance our pipeline and progress the field by leveraging our Centyrin platform to enable efficient and tissue-targeted delivery of siRNA. 

“This funding enables us to continue to pursue our mission and bring new medicines to patients who desperately need more effective treatments.”

The company focuses on developing tissue-targeted genetic therapies using the Centyrin protein technology platform. 

It is also working with industry partners to utilise the platform for tissue-specific targeting of therapeutics to treat a wide range of diseases.

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.