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Industry
Healthcare
Company Type
Biotechnology/Biopharmaceuticals
Size
$6-21 Million
Investment Type
Equity
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Tamara Williams | Managing Director |
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twilliams@castleplacement.com |
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(385) 600-1871 |
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Gary Levy | Managing Director |
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glevy@castleplacement.com |
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(516) 457-0104 |
Overview
ERAD Therapeutics is a pre-clinical stage biotechnology company focused on developing treatments for rare genetic diseases, combining gene therapy and biomolecular technologies to address limitations in current rare disease treatments.
Raising $6M–$21M in Series A equity capital to develop its lead gene therapy programs, initially focused on Duchenne Muscular Dystrophy (DMD) and its proprietary mCT biomolecule platform.
Proprietary gene therapy platform designed to deliver the full dystrophin gene
Novel gene-agnostic biomolecular platform (mCT) addressing unmet needs across multiple rare diseases, including those requiring therapies capable of crossing the blood-brain barrier
Market timing bolstered by recent challenges in DMD therapies and demand for improved treatment modalities
Patent protection expected through 2044 and beyond
Orphan Drug Designation in Gaucher Disease
Problem
Many rare genetic diseases remain untreated or poorly treated, creating substantial unmet medical demand.
The Limitation
- Most gene therapies for Duchenne MD deliver micro‑dystrophin, a truncated form of the dystrophin protein that does not fully restore function
- Viral vector technologies have payload size limitations preventing delivery of the full dystrophin gene and often posing severe safety concerns
- Thousands of rare genetic diseases (RGDs) currently have limited or no therapeutic options
The Patient Impact
- Neurological symptoms of rare genetic diseases remain largely untreated due to inability of existing treatment to cross the blood‑brain barrier
- Enzyme Replacement and Substrate Reduction therapies are ineffective for Central Nervous System (CNS) symptoms, leaving major unmet clinical needs
Solution – Breakthrough Gene Therapy Technology
Full DMD Gene Therapy
- Designed with potential to deliver large genetic payloads, such as the full dystrophin gene, without viral vectors
- Aims to overcome viral vector payload and immunogenicity limitations
- Potential to improve therapeutic outcomes relative to truncated dystrophin protein approaches
- Multiple disease opportunities in Rare Genetic Diseases
Solution – Breakthrough mCT Biomolecular Technology
mCT Biomolecule
- mCT is gene-agnostic
- Mechanism-based, efficacy is independent of gene mutation
- Numerous disease targets provided mutated protein retains partial function
- Intranasal or gene therapy administration options
- Designed to restore functional proteins with potential application across a wide range of genetic disorders
Specific Risks
- Execution Risk: Ability to scale clinical development and secure subsequent large financings
- Competitive Risk: Presence of well-capitalized competitors with advanced platforms and marketed products
- Scientific Risk: Potential pre-clinical failures given early-stage biotechnology efforts
- Regulatory Risk: Potential for changes in regulatory guidance given present instability in the regulatory environment
- Clinical Risk: Potential for failure to meet key endpoints in clinical development
- Financial Risk: Current limited capital and operating losses raise going concern considerations
- May not be able to retain key talent
- Risk of not achieving an exit strategy
- Private securities are speculative, illiquid, and carry significant risk including total loss of investment
