Asceragen is a biopharmaceutical company focused on developing therapies for patients with rare diseases. Every day, Aceragen strives for engagement with patients as key stakeholders in the process of developing and bringing to market treatments with transformative potential

OUR WORK

The team at Aceragen has a deep commitment to respect, innovation, efficiency and integrity as we work to serve patients with acid ceramidase deficiency, and to further the interests of the broader rare disease community. We undertake the journey together and look to learn from all stakeholders in order to reach our common goal of making truly transformative treatments available to patients who need and deserve them. For us, this means working together for solutions that work in the real world and over the long run.

PRINCIPALS

John Taylorw

John Taylorw

John Taylorw

John Taylorw

HIGHLIGHTS

Diversified Development Platform With High Value Programs Across Stages.

  • Targetting Series A of $35M to enable multiple clinical milestones
  • Lead Program targets significant commercial opportunity for untreated rare diseases with rapid path to approval
  • Single registration study agreed with FDA/EMA - Approval in 2003
  • Potential expansion into additional mechanically aligned rare indicatons
  • Potential approval in USA and EU in 3 years for untreated patient population with severe disease(Orphan & Fast-Track designations granted)
  • Initial disease market anticipated to be $200M-$300M; No known competetion
  • Related Indications expand peak sales opportunity substantially
  • Additional 1-2 IND fillings within 24 months from current pipeline
  • Clinical or near-clinical acquisitions under evaluation have game-changed potential for patients, attainable under attractive terms
  • Management team, Board and Advisors with broad industry expertise in product development, technical operations, product supply, business and corporate development and capital sourcing

ADVISORS & CONSULTANTS

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ANTHONY HICKEY, PHD

Product Development

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GREG LANDES, PHD

Antibody Development

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BRIAN JACKEY

Technical Operators

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DAVID MOOREY, PHD

Metabolic Disease

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BERNADETTE KEANE, PHD

Product Development, Cell and Gene Therapy

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RAYREILLY

Clinical Development

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CARL KARUS,MD

Clinical, Regulatory

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DAVID SCOTT, PHD

Product Development, Cell and Gene Therapy

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