ACG-801 Enzyme
Replacement Therapy

The team at Aceragen is working rapidly with our lead program ACG-801 to initiate a clinical trial for investigational enzyme replacement therapy treatment of acid ceramidase deficiency presenting as Farber disease. We also plan to develop treatments for additional rare diseases with high unmet need.

Farber disease is a rare metabolic disease that usually has four hallmark symptoms that appear in very early childhood.

  • Joint pain or contractures
  • Painful and swollen joints
  • Bumps or nodules under the skin
  • Increasing weak or hoarse voice

These symptoms can appear at any age and any order. They can also vary in severity and how quickly they progress, making it hard to diagnose. Most patients are initially misdiagnosed as having juvenile idiopathic arthritis

RTV - 801

Enzyme Replacement Therapy for Farber Disease

At Aceeragen we are developing a unique therapy for Faber’s patients to help resolve the symptoms and progression of this debilitating disease. The following are three depictions of our goal of moving patients with severe life threating forms of Farber’s to mitigating symptoms to return to

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RAPIDLY PROGRESSIVE (SEVERE)

Early onset and rapid progression if not treated results in severe pain and profound inflammation.

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MODERATE TO SLOW PROGRESSION

First symptoms in early childhood vary in severity with mild joint pain to debilitating nodules, and is often misdiagnosed with juvenile idiopathic arthritis.

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POTENTIAL TO RETURN TO A FUNCTIONAL LIFE

It’s our goal at Aceragen to provide therapies for rare disease patients allowing them to have some semblance of a normal life

ANTI - ASPROSIN MAB

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ENAC PEPTIDES

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