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Krystal Biotech (NASDAQ:KRYS) Portfolio, Stock and Financial Analysis

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Precision Biosciences(Nasdaq: DTIL) Portfolio, Stock and Financial Highlights

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Stoke Therapeutics (Nasdaq: STOK) Portfolio, Stock & Financial Highlights

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IONIS Pharma (Nasdaq: IONS) Portfolio, Stock and Financial Highlights

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Step-wise Procedure to Calculate Screening Cut Point (Parametric and Robust Parametric Method) for Anti-Drug Antibody Assay

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REGENXBIO (Nasdaq: RGNX)Gene Therapy Portfolio, Stock and Financial Highlights

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FDA AND EMA Approved OXLUMO™ (Lumasiran) for the Treatment of Primary Hyperoxaluria Type 1

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AAV5-RPGR Gene Therapy for RPGR-Associated X-Linked Retinitis Pigmentosa- Janssen (Johnson & Johnson) & MEIRAGTx

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Inclisiran (siRNA) for the Treatment of Hypercholesterolemia: PDUFA Date Set as December 2020

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ARU-1801 (Aruvant), Investigational Gene Therapy for Treatment of Sickle Cell Disease: Regulatory Update

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Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A

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Elivaldogene autotemcel (Eli-cel) for treatment of Adrenoleukodystrophy (ALD): Regulatory Update

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Eladocagene exuparvovec for the treatment of Aromatic L-amino acid decarboxylase (AADC): Regulatory Update

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Lenadogene nolparvovec LUMEVOQ® for the treatment of Leber Hereditary Optic Neuropathy (LHON): Regulatory Update

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TEGSEDI (Inotersen) Injection for Treatment of Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis

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AAV5-PAH Gene Therapy Clinical Study for Subjects With Phenylketonuria: Update

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Alnylam Pharmaceuticals (NASDAQ: ANLY)Portfolio:Approval of OXLUMO™ (lumasiran) and Expected approval of LEQVIO® (inclisiran) By End of 2020

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Bamlanivimab Neutralizing IgG1 Monoclonal Antibody for Treatment of COVID-19

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Exon Skipping EXONDYS 51 (eteplirsen) for Treatment of Duchenne Muscular Dystrophy

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