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.

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. 

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

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.

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