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Gene therapy company 4D Molecular Therapeutics raises $70m in Series C round

Gene therapy company 4D Molecular Therapeutics raises $70m in Series C round

California-based gene therapy company 4D Molecular Therapeutics (4DMT), which is focused on adeno-associated virus (AAV) gene therapy vector discovery and product development, has raised $70 million through a Series C financing round. The Series C financing round was led by Viking Global Investors. It also saw participation from new investors such as Amzak Health, Casdin […]

Roche signs $2.85bn deal with Sarepta for SRP-9001 DND gene therapy

Roche has signed a licensing deal worth up to $2.85 billion with Sarepta Therapeutics for the exclusive ex-US commercial rights to the latter’s SRP-9001 (AAVrh74.MHCK7.micro-dystrophin), an investigational gene therapy for Duchenne muscular dystrophy (DMD). As per the agreement terms, Sarepta Therapeutics will get an upfront payment made up of $750 million in cash and $400 […]

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Astellas Pharma to acquire US gene therapy company Audentes Therapeutics

Astellas acquisition of Audentes: Japanese pharma company Astellas Pharma has agreed to acquire US gene therapy company Audentes Therapeutics in an all-cash deal worth about $3 billion, as per pharma acquisition news. Based in California, Audentes Therapeutics calls itself a clinical-stage company focused on developing a portfolio of Adeno-associated virus (AAV)-based genetic medicines for the […]

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Ferring, Blackstone invest $570m in nadofaragene firadenovec gene therapy

Ferring Pharmaceuticals, a Swiss specialty biopharmaceutical group, has teamed up with private investment platform Blackstone Life Sciences to jointly invest more than $570 million in nadofaragene firadenovec (rAd-IFN/Syn3), an investigational gene therapy. Currently, nadofaragene firadenovec is in late-stage development for the treatment of patients with high-grade, Bacillus Calmette-Guérin (BCG) unresponsive, non-muscle invasive bladder cancer (NMIBC). […]

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Cellectis begins MELANI-01 clinical trial for UCARTCS1 in multiple myeloma

Cellectis said that it has dosed the first patient in a phase 1  study – the MELANI-01 clinical trial to evaluate UCARTCS1, an allogeneic off-the-shelf CAR-T product candidate for the treatment of relapsed/refractory multiple myeloma (R/R MM). The French gene therapy company began the phase 1 dose-escalation study after getting clearance from the US Food […]

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Avrobio gets FDA orphan status for AVR-RD-02 in Gaucher disease

US gene therapy company Avrobio has secured orphan-drug designation for AVR-RD-02, its investigational gene therapy, for the treatment of Gaucher disease, from the US Food and Drug Administration (FDA). AVR-RD-02 is made up of the patient’s own hematopoietic stem cells, which are modified genetically to express glucocerebrosidase (GCase), an enzyme that is deficient in Gaucher […]

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Castle Creek to acquire cell and gene therapy company Fibrocell

Castle Creek acquisition of Fibrocell : Castle Creek Pharmaceutical Holdings (CCP Holdings) has agreed to acquire Fibrocell Science, a cell and gene therapy company engaged in developing autologous cell-based therapies for skin and connective tissue disorders. Financial terms of the deal were not disclosed, as per the latest pharma industry news. CCP Holdings is a […]

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US gene therapy company Renovacor raises $11m for BAG3 gene therapy

US gene therapy company Renovacor, which is engaged in developing BAG3 gene therapy and other gene therapies for cardiovascular disease, has raised $11 million through a Series A financing round led jointly by Novartis Venture Fund, Broadview Ventures, and BioAdvance. New Leaf Venture Partners and Innogest Capital also joined the Series A funding round. The […]

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Apic Bio bags FDA orphan designation for ALS SOD1 gene therapy APB-102

US gene therapy company Apic Bio said that APB-102, its ALS SOD1 gene therapy, has been granted orphan drug designation by the US Food and Drug Administration (FDA). APB-102 is expected to shortly enter into clinical development for the genetic disorder – SOD1 amyotrophic lateral sclerosis (ALS). The orphan designation is given to novel drugs […]

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Biogen to acquire UK gene therapy company Nightstar Therapeutics for $800m

Biogen acquisition of Nightstar Therapeutics : US biotech company Biogen has signed a deal of $800 million to acquire Nightstar Therapeutics, a British clinical-stage gene therapy company, as per the latest pharma acquisition news. Nightstar Therapeutics primarily focuses on the developing adeno-associated virus (AAV) treatments for inherited retinal disorders. Biogen acquisition of Nightstar Therapeutics According […]

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