BEIJING – Gene therapy startup Hui-Gene Therapeutics Ltd. Co., of Shanghai, said it secured more than ¥100 million (US$14 million) in a series A financing round to develop a safer gene therapy to treat genetic diseases caused by single-base mutations.
Huntington’s disease is a fatal hereditary disease that results in the progressive breakdown of nerve cells in the brain. It erodes a person’s physical and mental abilities, usually beginning in their 30s and 40s, and to date has no cure. Now Austin-based Asuragen Inc. is joining forces with Wave Life Sciences USA Inc., of Cambridge, Mass., to develop companion diagnostics (CDx) for Wave’s investigative allele-selective therapeutic programs targeting the genetic cause of the disease.
The mantra for a while has been that it's not good enough to get your drug approved, you've got to get it payed for, too. For gene therapy treatments, the payment is more complex, and you can add manufacturing and supply chain logistics to the list of challenges.
As Wall Street ponders how pricing and reimbursement for gene therapies might shake out, companies in the field march intrepidly on, among them Orchard Therapeutics Inc., which early this month rolled out heartening results from an ongoing proof-of-concept trial evaluating the safety and efficacy of OTL-203 for mucopolysaccharidosis type I (MPS-I), a space that's heating up.
DUBLIN – Gyroscope Therapeutics Ltd. raised £50.4 million (US$60.5 million) to further development of GT-005, its clinical-stage gene therapy for treating dry age-related macular degeneration (AMD), and to advance a second-generation of the subretinal delivery system it gained through its recent merger with Orbit Biomedical Ltd.
SEOUL – South Korea's investors have become very interested in the global cell and gene therapy market. Licensing and M&A deals in the field have been active – a good sign for Korean biopharma firms eager to tap in.
DUBLIN – Every technology shift has its winners and losers. Ireland, long a key location for small-molecule and biologics manufacturing, is now gearing up to ensure it is on the right side of the divide as the rollout of cell and gene therapies (CAGT) gathers momentum. There are no guarantees that the same technological and economic factors that contributed to its success during earlier eras of pharmaceutical and biopharmaceutical manufacturing will apply to the coming era of regenerative medicine. But the country's inward investment agency, IDA Ireland, is betting that its long-established relationships with the top tier of biopharma companies and its longstanding expertise in meeting FDA manufacturing requirements will help in the battle to win new investment projects.
TEL AVIV, Israel – With shares now listed in the U.S. and data from a pivotal trial for its key bladder cancer therapy coming soon, Anchiano Therapeutics Ltd. is poised to ride the wave of interest in gene therapy.
LONDON – With five expensive advanced therapies recently approved in Europe and a further five expected to get approval in the near term, it is vital the hurdles that delay and preclude access – and which have led to the commercial failure of earlier products – are removed.
In late May, Novartis AG's Avexis Inc. unit gained FDA approval for Zolgensma (onasemnogene neparvovec) to treat spinal muscular atrophy, and other companies are looking to follow suit developing drugs to treat a variety of diseases of the central nervous system (CNS).
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