Lilly to acquire US gene therapy company Prevail Therapeutics in $1bn deal

Lilly to acquire US gene therapy company Prevail Therapeutics in $1bn deal

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 […]

HMR59 : Janssen acquires rights to geographic atrophy treatment candidate from Hemera

HMR59 : Janssen acquires rights to geographic atrophy treatment candidate from Hemera

Geographic atrophy treatment : Janssen Pharmaceuticals has signed a licensing deal to acquire rights to HMR59, an investigational gene therapy for geographic atrophy, from clinical stage ocular gene therapy company Hemera Biosciences for an undisclosed price. HMR59 is intended to be given as a one-time, outpatient, intravitreal injection for helping in preserving vision in patients […]

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 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 […]

Astellas Pharma to acquire US gene therapy company Audentes Therapeutics

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 […]

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

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). […]

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

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 […]

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

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 […]

4D Molecular Therapeutics secures $90m in Series B to advance gene therapy innovations

4D Molecular Therapeutics secures $90m in Series B to advance gene therapy innovations

California-based 4D Molecular Therapeutics (4DMT), specializing in adeno-associated virus (AAV) gene therapy vector discovery and product development, has successfully raised $90 million through a Series B financing round. The round was led by Viking Global Investors, with participation from notable investors including ArrowMark Partners, Janus Henderson Investors, The Biotechnology Value Fund, MiraeAsset Financial Group, Pfizer […]