The Latest Breaking News from Gene Therapy

Oyster Point Pharma Inc (NASDAQ:OYST) will implement an operating expense streamlining plan to reduce employee and non-employee expenses (up to 50 roles) across the organization.  The company plans to continue to drive the launch of Tyrvaya with approximately 150-200 field-based sales resources. The plan is expected to reduce operating expenses by approximately $6…

UniQure N.V. (NASDAQ:QURE) announced safety and biomarker data from 10 patients enrolled in the low-dose cohort of the ongoing Phase 1/2 trial of AMT-130 gene therapy for Huntington's disease.  Six of the ten enrolled patients received AMT-130, and four received an imitation surgical procedure in this randomized, blinded clinical trial in the U.S.…

The FDA signaled in briefing documents released that it may approve Bluebird bio Inc's (NASDAQ:BLUE) beti-cel gene therapy to treat β-thalassemia for those who require regular blood transfusions. The Prescription Drug User Fee Act (PDUFA) goal dates for beti-cel and eli-cel are August 19 and September 16. Bluebird bio's blood disorder treatment demonstrates "clinically meaningful"…

SVB Leerink initiated Selecta Biosciences Inc (NASDAQ:SELB) coverage with an Outperform rating and a price target of $7. The analysts view ImmTOR's approach to mitigate ADAs as de-risked by early clinical data from SEL-212 and believe that the ability to tame the immune response bodes well for ImmTOR's clinical potential in combination with…

Last week, PTC Therapeutics Inc's (NASDAQ:PTCT) Upstaza (PTC-AADC; AAV2-based DDC gene therapy) received a CHMP opinion for approval under exceptional circumstances, says Raymond James. It maintains Outperform rating on the stock. The CHMP recommended Upstaza for AADC deficiency (AADCD) patients aged 18 months and older with a severe phenotype. According to the analysts, with the…

CRISPR is a versatile tool for editing genomes and has recently been approved as a gene therapy treatment for certain blood disorders.