- In November 2024, PTC Therapeutics announced FDA accelerated approval for its gene therapy targeting AADC deficiency, marking the first U.S.-approved gene therapy administered directly to the brain. This milestone strengthens PTC's position in innovative gene therapies and expands its treatment portfolio for rare disorders
- In July 2024, Genezen, announced its acquisition of uniQure’s commercial gene therapy operations in Lexington, Massachusetts, for USD 25 Billion on July 1, 2024. This acquisition enhances Genezen's capabilities in commercial gene therapy production, positioning it for growth in the expanding gene therapy market
- In May 2024, Merck has agreed to acquire Mirus Bio, a Madison, Wisconsin-based leader in transfection reagents, for USD 600 Billion. Mirus Bio's solutions, including TransIT-VirusGEN, are crucial for producing viral vectors in cell and gene therapies. This acquisition strengthens Merck's position in the cell and gene therapy market by expanding its portfolio of innovative tools for genetic material delivery
- In May 2024, Charles River Laboratories has launched Modular and Fast Track frameworks for viral vector technology transfer, enabling accelerated process transfers to its Maryland-based center of excellence within nine months. This initiative enhances the company's efficiency in supporting gene therapy development and strengthens its position as a leading CDMO
- In March 2022, researchers at Mass Eye and Ear unveiled a promising method for delivering genetic material to the inner ear of non-human primates, which could lead to potential treatments for hearing loss and vestibular disorders. Their findings, published in Nature Communications, highlight the success of combining the synthetic adeno-associated virus (AAV) vector Anc80L65 with an innovative surgical technique known as transmastoid posterior tympanotomy to effectively target the inner ear
