Company profile: Orphazyme
1.1 - Company Overview
Company description
- Provider of biopharmaceutical R&D for novel therapeutics targeting lysosomal storage diseases and other genetic disorders; conducts clinical trials, offers early access programs for investigational drugs, and delivers safety reporting/pharmacovigilance. Developed arimoclomol for neurodegenerative diseases, including Niemann Pick disease type C (sold to KemPharm Denmark A/S).
Products and services
- Biopharmaceutical Research and Development: Engineers novel therapeutics for genetic disorders, focusing on lysosomal storage diseases with activities and investments grounded in founders’ Danish Cancer Society discoveries
- Research-grade
- Clinical Trials: Conducts studies to evaluate efficacy and safety of compounds for treating genetic disorders, supporting advancement of lysosomal storage disease therapies through patient investigations
- Clinical-stage
- Early Access Program: Delivers access to investigational drugs outside clinical trials for patients with serious conditions, enabling treatment availability while formal studies progress
- Investigational
Key contacts
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Financial details
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1.2 - Competitors and similar companies to Orphazyme
Chord Therapeutics
HQ: Switzerland
Website
- Description: Provider of clinical-stage biopharmaceutical drug development for rare, life-threatening and severely disabling diseases, advancing lead candidate CRD1 under orphan drug designation for neuromyelitis optica spectrum disorders (NMOSD) and myasthenia gravis (MG).
- 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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Perlara
HQ: United States
Website
- Description: Provider of precision drug discovery services: drug repurposing screening to identify potential treatments and advise on experimentation and clinical translation; CureMap disease analyses with literature review, ongoing research, expert interviews, and partner identification; scientific advisory for reviews, client meetings with scientific partners, and translation guidance; and Guided Cures planning with researchers and clinicians.
- 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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ProFactor Pharma
HQ: United Kingdom
Website
- Description: Provider of recombinant blood factors for haemophilia, including a low-cost, GMP-optimized recombinant human Factor VIII (rhFVIII) for Haemophilia A. Offers a patent-protected cell line and production process delivering higher yields and lower costs, and collaborates with Bio Farma Persero on scale-up, clinical trials planned to commence in 2024, and commercialization.
- 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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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
- Employees: ●●●●●
- Total funding raised: $●●●m
- Backers: ●●●●●●●●●●
- Acquisitions: ●●
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Actio Biosciences
HQ: United States
Website
- Description: Provider of rare disease drug discovery and development, with a small molecule TRPV4 program for Charcot-Marie-Tooth disease type 2C and serious bone diseases; a proprietary Rare Disease Target Atlas for target identification and function; a partnership with The Jackson Laboratory to create transgenic models for target evaluation and phenotype analysis; and upcoming programs for genetic epilepsies and genetic skin conditions.
- Rationale: This competitor operates in a highly similar market segment with overlapping features.
- Company type: Private company
- Employees: ●●●●●
- Total funding raised: $●●●m
- Backers: ●●●●●●●●●●
- Acquisitions: ●●
View full Actio Biosciences company profile →
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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 Orphazyme
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3. Investors and private equity firms
3.1 - Buyout funds investing in similar companies to Orphazyme
2.2 - Growth funds investing in similar companies to Orphazyme
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4 - Top valuation comps for Orphazyme
4.2 - Public trading comparable groups for Orphazyme
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