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).

With the 2012 BIO International Convention behind us and the Fourth of July signaling summer vacation season in earnest, thoughts turn to visions of sun, sand and afternoons lounging in a deck chair. Again this year, BioWorld polled biotech execs, industry analysts and our own staff to construct a diverse list of titles for your reading pleasure. Whether your tastes run to historical intrigue or the classics, professional development or science fiction, you’ll find something of interest on our sixth annual list. Fiction: 16th Century England to 20th Century Los Angeles Diego Miralles, head of Janssen West Coast Research Center...

If you can’t see the wood for the trees the common sense response is to do a little thinning and let the light shine through. But for the bogged-down-in-bureaucracy European Medicines Agency (EMA), the response last week to the need to increase transparency and streamline its procedures was to set up an expert committee to investigate the activities and operations of its expert committees. I don’t imagine this is a cynical move by the recently installed head of EMA, Guido Rasi, to kick complaints about a lack of transparency and failure to listen to the needs of patients into the...

Reading last weekend’s Wall Street Journal review of “The Forever Fix: Gene Therapy and the Boy Who Saved It” I was struck by an anecdote. It’s about an interview the reviewer did with a scientist who works in the field of neuroprostheses, and that scientist’s refusal to talk about the possible practical applications of his work, because, he said, “false hope is a sinful thing.” Really? To me, it seems like an inevitable part of hope is that it might be false. To illustrate, I don’t hope that my neighbors will be nice to me, because it’s a sure thing....