The acceptance by the FDA of Prevail Therapeutics Inc.’s IND for the one-time, fast-tracked gene therapy PR-006 provided hope for 50,000 to 60,000 people in the U.S beset by frontotemporal dementia with the GRN mutation (FTD-GRN), and the New York-based company is moving ahead with a phase I/II experiment called Proclaim.
DUBLIN – Genespire Srl, a Milan-based startup led by gene therapy pioneer Luigi Naldini, is the third company to receive funding from Sofinnova Partners’ Italian €108 million (US$117.5 million) gene therapy fund, the Sofinnova Telethon Fund.
HONG KONG – Daiichi Sankyo Co. Ltd., Mitsubishi UFJ Capital Co. Ltd. and Nagoya Institute of Technology have kicked off research on a joint project for a gene therapy that could be applied to the restoration of vision.
Avectas Ltd., of Dublin, brought in a $20 million series C financing, ratcheting the total equity investment in the company to $40 million. Privately held Avectas, formed in 2012 as a spin-out from Ireland’s Maynooth University, said it plans to accelerate the clinical translation and commercial scale-up of its cell engineering technology and expand its staffing in Ireland.
It has been more than 200 years since British doctor James Parkinson first identified the symptoms of a condition that he termed shaking palsy; unfortunately, there is still no cure to the disease that carries his name.
LONDON – These are hardly times for a fanfare, but this month saw the unveiling of a new name in bioprocessing, following the formal closing of the $21.4 billion sale of GE Healthcare’s Life Sciences to Danaher Corp. The business, now renamed Cytiva, has turnover of $3.3 billion, nearly 7,000 employees and operations in 40 countries. More than 75% of FDA-approved biologic drugs use its products in their manufacture.
The allure of gene therapy was proved yet again as Waltham, Mass.-based Affinia Therapeutics Inc. bagged an oversubscribed $60 million series A financing to boost the push for drugs to benefit people affected by muscle and central nervous system conditions.
Cambridge, Mass.-based Elevatebio LLC has triple-pronged plans for its $170 million series B round, which the company said will be directed toward manufacturing cell and gene therapies, enabling new technology platforms, and pursuing therapeutics.
During the conference call on earnings in March, CEO Dror Harats told investors that “the most important thing” about VBL Therapeutics Ltd.’s then-upcoming analysis of interim phase III data with gene therapy VB-111 (ofranergene obadenovec) is that it was “designed in a way that will enable us to tell the market if we are at least as good as what we've seen” in the phase II experiment.
Strongly favorable six-month data disclosed on Jan. 9 by Applied Genetic Technologies Corp. (AGTC) from its ongoing phase I/II program with an adeno-associated virus (AAV) gene therapy for X-linked retinitis pigmentosa (XLRP) did more than provide a whopping stock boost.
