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The Latest Breaking News from Gene Therapy

Sarepta Reports One Heart Inflammation Case In Duchenne Gene Therapy Trial

Sarepta Therapeutics Inc (NASDAQ:SRPT) shared new functional data across multiple studies from the SRP-9001 (delandistrogene moxeparvovec) program for Duchenne muscular dystrophy. SRP-9001 is an investigational gene therapy under development in partnership with Roche Holdings AG (OTC:RHHBY). Results from Cohort 1 demonstrated a 3.8-point improvement and 3.2-point improvement on the Duchenne functional scale 52 weeks after…

Why Precigen Shares Are Gaining Today

Why Precigen Shares Are Gaining Today

Precigen Inc (NASDAQ:PGEN) has agreed to sell its subsidiary Trans Ova Genetics, an animal reproductive technologies company, to URUS, a holding company with cooperative and private ownership. The deal consideration includes $170 million in upfront cash and up to $10 million earn-out based on the performance of Trans Ova in 2022 and 2023. …

Oyster Point Outlines Restructuring Plan With Savings Of Up To $48M In 2023

Oyster Point Outlines Restructuring Plan With Savings Of Up To $48M In 2023

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…

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UniQure Shares Are Up After Low-Dose Cohort Data From Huntington's Gene Therapy Trial

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.…

BridgeBio Pharma Posts Promising Data From Gene Therapy Trial In Ultra-Rare Disease

BridgeBio Pharma Inc (NASDAQ:BBIO) announced promising pharmacodynamic data from the first two participants dosed in CANaspire Phase 1/2 trial of BBP-812, gene therapy for Canavan disease. Canavan disease is an ultra-rare inherited disorder that damages the ability of nerve cells (neurons) in the brain to send and receive messages. Data from the first…

Mizuho Comments On UniQure's Upcoming Low-Dose Huntington's Gene Therapy Trial Data

The much-awaited one-year data for the entire low-dose cohort for UniQure NV's (NASDAQ: QURE) AMT-130 gene therapy in Huntington's disease is expected at any time.

FDA Sounds Positive On Bluebird's One Gene Therapy But Doubts Another

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"…

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SVB Leerink Sees Over 600% Upside For This Penny Biotech Stock

SVB Leerink Sees Over 600% Upside For This Penny Biotech Stock

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…

Raymond James Says PTC Therapeutics' CHMP Backing Is 'Under Exceptional Circumstances' What's Next

Raymond James Says PTC Therapeutics' CHMP Backing Is 'Under Exceptional Circumstances' What's Next

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…

How does CRISPR work?

How does CRISPR work?

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

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Jennifer Doudna

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