CSL Behring, a US-based biotechnology company, has received approval for its Hemgenix (etranacogene dezaparvovec-drlb) from the US Food and Drug Administration (FDA) as a one-time gene therapy for hemophilia B in adults. Hemophilia B is a bleeding disorder resulting from a single gene defect, which causes inadequate production of factor IX. Produced mainly by the […]
Genprex has dosed the first patient in the Acclaim-1 phase 1/2 clinical trial of REQORSA immunogene therapy candidate in combination with Tagrisso (osimertinib) in a certain patient population having late-stage non-small cell lung cancer (NSCLC). The combination of REQORSA and Tagrisso is being evaluated in the trial in late-stage NSCLC patients with activating epidermal growth […]
Oxford Biomedica, a UK-based gene and cell therapy company, has agreed to form a joint venture (JV) to undertake an adeno-associated virus (AAV) manufacturing and innovation business in the US with Homology Medicines. The joint venture has been named — Oxford Biomedica Solutions. As per the terms of the agreement, Oxford Biomedica will pay $130 […]
Novartis has agreed to acquire Gyroscope Therapeutics, a UK-based ocular gene therapy company, in a deal worth up to $1.5 billion. As per the terms of the deal, the Swiss pharma giant will pay $800 million upfront followed by up to $700 million in potential additional milestone payments. Through the deal, Novartis will gain access […]
Ocugen said that the US Food and Drug Administration (FDA) has accepted its investigational new drug application (IND) to launch a first-in-human clinical trial of the OCU400 (AAV-NR2E3) gene therapy candidate for the treatment of retinitis pigmentosa caused by genetic mutations found in NR2E3 and Rhodopsin. The IND acceptance by the FDA allows the US-based […]
Selecta Biosciences said that the US Food and Drug Administration (FDA) has imposed a clinical hold on its phase 1/2 clinical trial of SEL-302 gene therapy for the treatment of methylmalonic acidemia (MMA). SEL-302 consists of MMA-101 plus ImmTOR immune tolerance platform. The US-based biotech company said that it has been sent a letter from […]
Vivet Therapeutics, a biotech company backed by Pfizer, has been given fast track designation by the US Food and Drug Administration for its gene therapy candidate VTX-801 for the treatment of Wilson Disease. Wilson Disease is a rare, genetic disorder that lessens the ability of the liver and other tissues in controlling copper levels, thereby […]
Charles River Laboratories International has signed an all-cash deal worth $292.5 million to acquire Vigene Biosciences, a US-based gene therapy contract development and manufacturing organization (CDMO) that offers viral vector-based gene delivery solutions. Vigene Biosciences also stands to earn up to $57.5 million in contingent additional payments based on its future performance. The acquisition is […]
Eli Lilly and Company (Lilly) has completed its previously announced deal worth up to $1.04 billion of US gene therapy company Prevail Therapeutics. The US pharma giant said that the deal gives it a new modality for drug discovery and development. Furthermore, it will extend its research efforts via the creation of a gene therapy […]
Eli Lilly and Company (Lilly) has signed a deal worth up to $1.04 billion to acquire Prevail Therapeutics, a US gene therapy company engaged in developing disease-modifying AAV9-based gene therapies for neurodegenerative diseases. According to the US pharma giant, the acquisition will set up a new modality for drug discovery and development. It will also […]