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Enzyme therapy

Ultragenyx’s Gene Therapy Ameliorates Pediatric Neurodegenerative Disorder
Drug Discovery

Ultragenyx’s Gene Therapy Ameliorates Pediatric Neurodegenerative Disorder

Rare Disease Therapeutics: Accelerating Development and Improving Access
Drug Discovery

Rare Disease Therapeutics: Accelerating Development and Improving Access

One-Pot Method for Generating Orally Available Cyclic Peptide Drug Candidates
Drug Discovery

One-Pot Method for Generating Orally Available Cyclic Peptide Drug Candidates

Gene Therapy for ADA-SCID Still Benefitting Patients after Two to Three Years
Genome Editing

Gene Therapy for ADA-SCID Still Benefitting Patients after Two to Three Years

Sangamo Shares Plunge after Interim Results for MPS Candidates Fall Short of Clinical Benefit
Featured

Sangamo Shares Plunge after Interim Results for MPS Candidates Fall Short of Clinical Benefit

Gene Therapy for Rare Diseases
Insights

Gene Therapy for Rare Diseases

FDA Approves Ultragenyx Enzyme Replacement Therapy for MPS VII
Industry News

FDA Approves Ultragenyx Enzyme Replacement Therapy for MPS VII

Nestlé Health Science, Codexis to Develop PKU Enzyme in Protein Platform Partnership
Bioprocessing

Nestlé Health Science, Codexis to Develop PKU Enzyme in Protein Platform Partnership

Erytech, Queen’s Partner to Develop Red Blood Cell-Encapsulated Enzyme Therapy
Drug Discovery

Erytech, Queen’s Partner to Develop Red Blood Cell-Encapsulated Enzyme Therapy

Rubius Therapeutics Closes $120M Financing, Plans Trials Next Year
Cancer

Rubius Therapeutics Closes $120M Financing, Plans Trials Next Year

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