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.

Something of a duel may be shaping up between Menlo Park, Calif.-based Adverum Biotechnologies Inc. with ADVM-022, the phase I gene therapy candidate for wet age-related macular degeneration (AMD) and Regenxbio Inc., of Rockville, Md., with a similar candidate.

Tenaya Therapeutics Inc. CEO Faraz Ali told BioWorld that the company, which raised $92 million in a series B round, has programs from three cardiac platforms "stacked on top of each other" and wants to enter the clinic by the end of 2021 "with at least one of the multiple projects we're advancing." The South San Francisco-based firm wanted not to "let resources be the barrier" as to which goes first, he said. "We wanted the science to dictate that."

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.

According to a new report from the Alliance for Regenerative Medicine, there are a whopping 932 regenerative medicine companies worldwide that are in the process of developing 440 gene therapies, 587 cell therapies and 125 tissue engineering/biomaterials products.

While many companies use viruses and viral vectors to deliver gene therapy and to modify cells for CAR T treatments, others have shunned adeno-associated viruses (AAV) and lentiviral vectors for other methods to deliver DNA and RNA into the cells.

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.

In late June, when Pfizer Inc. unveiled the first phase Ib data, mixed safety signal and all, for its Duchenne muscular dystrophy (DMD) gene therapy, PF-06939926, investors in Sarepta Therapeutics Inc. as well as Solid Biosciences Inc. watched with particular interest. The latter firm seems none the worse for wear, though, raising $60 million in a private placement.

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).