Company profile: Zacharon Pharmaceuticals
1.1 - Company Overview
Company description
- Provider of glycobiology-driven therapeutics targeting glycans for lysosomal storage diseases, including MPS I, II, and III, ganglioside inhibitor programs, and other lysosomal storage disease programs, plus Sensi-Pro clinical diagnostic analyzing glycans for disease diagnosis and monitoring.
Products and services
- MPS I, II, and III Program: Custom-engineered therapeutic program that modulates glycans involved in mucopolysaccharidoses types I, II, and III to impact lysosomal storage disease biology
- Ganglioside Inhibitor Program: Mechanism-based drug development initiative aimed at inhibiting gangliosides to treat certain lysosomal storage diseases by targeting glycan pathways
- Other Lysosomal Storage Diseases Program: Research-grade program targeting additional lysosomal storage diseases through glycan modulation, leveraging unique glycobiology expertise to develop an entirely new class of human therapeutics.
Key contacts
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Financial details
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1.2 - Competitors and similar companies to Zacharon Pharmaceuticals
Aro Biotherapeutics
HQ: United States
Website
- Description: Provider of tissue-targeted genetic medicines leveraging Centyrins, a proprietary small engineered protein platform, for targeted delivery to specific cells. Develops oligonucleotide-based therapeutics that modulate gene expression, including ABX1100, a Centyrin-siRNA conjugate targeting CD71 and Gys1 mRNA for Pompe disease to reduce muscle glycogen.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
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Evox Therapeutics
HQ: United Kingdom
Website
- Description: Provider of engineered exosome-based delivery and therapeutics via its DeliverEX platform to modify exosomes, load drugs, and target delivery to specific organs. Solutions include treatments for rare diseases, exosome-enabled gene therapy (AAV cloaking), gene editing with CRISPR, RNA therapeutics, and precision targeting to the brain and central nervous system.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
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- Total funding raised: $●●●m
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MBX Biosciences
HQ: United States
Website
- Description: Provider of preclinical-stage biotech therapies for rare endocrine diseases, developing investigational candidates including MBX 2109 for consistent parathyroid hormone exposure in hypoparathyroidism, MBX 1416 to prevent severe hypoglycemia in post-bariatric hypoglycemia, MBX 4291, a long-acting GLP-1/GIP receptor co-agonist for obesity, and the PEP Platform for precision endocrine peptide therapeutics.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
- Employees: ●●●●●
- Total funding raised: $●●●m
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Cabaletta Bio
HQ: United States
Website
- Description: Provider of cell therapy solutions for B cell-mediated autoimmune diseases, leveraging clinically-validated and FDA-approved CAR T cell technology. Pipeline includes CABA-201, an investigational CD19 CAR T in RESET Phase 1/2 trials, a proprietary CABA platform, and CAART therapies targeting disease-causing B cells, including DSG3-CAART and MuSK-CAART for pemphigus vulgaris and myasthenia gravis.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
- Employees: ●●●●●
- Total funding raised: $●●●m
- Backers: ●●●●●●●●●●
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Ultragenyx
HQ: United States
Website
- Description: Provider of therapeutics for rare and ultra-rare diseases, including CRYSVITA for XLH, MEPSEVII for MPS VII, and DOJOLVI for LC-FAOD. Develops investigational therapies such as UX143 (setrusumab) for osteogenesis imperfecta and GTX-102 for Angelman syndrome. Offers UltraCare patient support with assistance for insurance coverage, financial support, and access to resources.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
- Employees: ●●●●●
- Total funding raised: $●●●m
- Backers: ●●●●●●●●●●
- Acquisitions: ●●
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2.M&A buyers
2.1 Potential strategic acquirers in the sector
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2.2 - Strategic buyer groups for Zacharon Pharmaceuticals
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3. Investors and private equity firms
3.1 - Buyout funds investing in similar companies to Zacharon Pharmaceuticals
2.2 - Growth funds investing in similar companies to Zacharon Pharmaceuticals
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4 - Top valuation comps for Zacharon Pharmaceuticals
4.2 - Public trading comparable groups for Zacharon Pharmaceuticals
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