Wall Street didn’t much like Uniqure NV’s decision to hold off reporting efficacy measures in the phase I/II trial with one-time gene therapy AMT-130 for the treatment of Huntington’s disease (HD), but safety findings proved encouraging and analysts held out hope.
LONDON – Newco Loqus23 Therapeutics Ltd. has closed a £7 million (US$9.4 million) extension of its seed round, providing the means to progress its lead small-molecule program in Huntington’s disease through lead optimization. The extended round, from the Novartis Venture Fund and the U.K. government-backed Dementia Discovery Fund, follows an initial seed investment of £4.5 million by DDF in 2019, that was not made public at the time.
Fresh off licensing a potential Parkinson’s disease therapy last month, Ipsen SA is again looking to build out its neurodegenerative disease portfolio, this time in Huntington’s disease and Angelman syndrome. An exclusive options deal with Exicure Inc. could bring it two new spherical nucleic acid (SNA) programs for the indications while delivering up to $1 billion for Exicure, plus potential royalties, on top of a $20 million up-front payment. The deal, Exicure's second major SNA collaboration after a hair-loss disorders deal with Abbvie Inc.’s Allergan, sent Exicure shares (NASDAQ:XCUR) up 34.1% to $1.81 on Aug. 2.
LONDON – The World Health Organization (WHO) is to set up a channel for confidential reporting of illegal, unregistered, unethical or unsafe human genome editing research, as part of a new governance framework it is proposing to develop.
Casualties continue to accrue in Huntington’s disease, but drug developers continue their work in the challenging, fatal genetic disorder that afflicts an estimated 2.71 per 100,000 people globally. In March, Basel, Switzerland-based Roche Holding AG pulled the plug on its phase III Generation HD1 study with the antisense therapy tominersen, licensed from Ionis Pharmaceuticals Inc., of Carlsbad, Calif., in a tie-up that dates back to the spring of 2013. Roche subsidiary Genentech Inc. said the move was based on an independent data monitoring committee's preplanned look at the drug's risk-benefit profile.
LONDON – Antibody prospecting specialist Alchemab Therapeutics Ltd. has raised £60 million (US$82.7 million) in a series A round, to take programs in neurodegenerative diseases and cancer into the clinic.
Shares of Ionis Pharmaceuticals Inc. (NASDAQ:IONS) fell 21.7% to $43.59 on March 23 after its longtime partner, Roche Holding AG, decided to stop dosing the antisense oligonucleotide tominersen in a global phase III manifest Huntington's disease (HD) study. Roche subsidiary Genentech Inc. said the move was based on an independent data monitoring committee's preplanned assessment of the drug's risk-benefit profile.
Small-molecule mRNA translation expert Anima Biotech Inc. has landed a significant new preclinical research deal with Takeda Pharmaceutical Co. Ltd., covering as many as six programs for genetically defined neurological diseases. The deal starts with $120 million in up-front and preclinical research milestone payments for Anima, but altogether the two-part collaboration could hold as much as $2.3 billion in clinical and commercial milestone payouts for the company, providing substantial support for its internal pipeline, Anima co-founder and CEO Yochi Slonim told BioWorld.
Bionaut Labs emerged from five years in stealth mode raising $20 million to develop Bionauts, microrobots designed to deliver therapies to treat brain disorders. The financing will support the company’s therapeutic program in glioma through preclinical development and further research and development in Huntington’s disease.
Bionaut Labs emerged from five years in stealth mode raising $20 million to develop Bionauts, microrobots designed to deliver therapies to treat brain disorders. The financing will support the company’s therapeutic program in glioma through preclinical development and further research and development in Huntington’s disease. Khosla Ventures led the financing with participation by Upfront Ventures, Revolution LLC, BOLD Capital Partners, and Compound.
