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Beam Therapeutics (Gene Editing) Portfolio, Stock and Growth

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

REGENXBIO 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

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

Vosoritide (BMN111) for the Treatment of Achondroplasia (Short Stature): Regulatory Update

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: Biomarin Withdrew MAA Application From EMA

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

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