Lysosomal Storage Disorders: Azafaros Takes Aim At Small Molecules

BioGeneration Ventures has given birth to a new European biotech, Azafaros, supplying seed funding for the development of a new generation of compounds to tackle rare metabolic disorders.  

SC1807_Seed-Funding_676427629_1200.jpg
Seed funding can be found to progress good ideas • Source: Shutterstock

Biotech and pharma companies have had some success in the past developing enzyme replacement therapies for lysosomal storage disorders such as Fabry disease and Gaucher disease, and a new European biotech company, Azafaros, spun out of Leiden University in the Netherlands, is aiming to build on that achievement by developing a new generation of agents for rare metabolic disorders.

The potential new class of therapeutic agents are small-molecule orally active, aza-sugar compounds which interfere with the metabolism of glycolipids...

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Scrip for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required

More from Financing

Finance Watch: A Mini-Surge In Follow-On Offerings Follows Positive Updates

 
• By 

Public Company Edition: A breakthrough designation, data and deals prompted big follow-on public offerings in late June and early July, including $402.5m for Cidara, $250.8m for Kymera and $230m for Dyne. Also, Revolution Medicines accessed up to $2bn from Royalty Pharma.

BerGenBio Plumps For Oncoinvent From Long List Of 50 Suitors

 
• By 

The merger with a fellow Norwegian biotech comes a few months after its lead asset, bemcentinib, bit the dust.

Global Pharma Interest In Korea Undeterred Despite Political Roller Coaster

 
• By 

Despite recent political turmoil, outside investor and corporate interest in South Korean biopharma innovation appears robust or even increasing.

In Brief: Sanofi Cements Support For Adagene

 
• By 

Deal Snapshot: The French drugmaker has been shifting its investments away from cancer to focus on immunology and inflammation but it continues to seek out interesting early-stage oncology assets.

More from Business

In Brief: Concentra Snaps Up Struggling IGM Biosciences In Latest Biotech Deal

 

Concentra Biosciences is acquiring struggling IGM Biosciences for $1.25 per share plus a contingent value right, with the deal expected to close in August 2025.

Quick Listen: Scrip’s Five Must-Know Things

 
• By 

In this week's episode: AbbVie’s big autoimmune CAR-T play; In Vivo CAR-T interest grows; China deal perspectives from BIO; global pharma interest in Korea undeterred; and a look at the biggest drug sales disappointments.

Second Quarter M&A Activity Looks Like More Of The Same, Mostly

 
• By 

The biopharma sector booked nearly the same number of acquisitions in each of the past two quarters, according to Evaluate data, but there are some encouraging signs in Q2.