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

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

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

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

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

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

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

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

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

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

Lisocabtagene maraleucel for the treatment of adults with relapsed or refractory (R/R) large B-cell lymphoma: Regulatory Update

Idecabtagene Vicleucel Ide-cel for the Treatment of Multiple Myeloma Regulatory Update

Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A

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

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

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

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

REPATHA (Evolocumab) PCSK9 Inhibitor for Treatment of Familial Hypercholesterolemia

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