Cell/Gene Tx channel feed

FDA of­fi­cials, ex­perts dis­cuss im­pact of Covid-19 on cell and gene ther­a­pies

While the FDA is still receiving investigational new drug applications (INDs) for cell and gene therapies, officials are concerned about the impact of the Covid-19 pandemic on clinical trials.

“It’s clear that COVID-19 has adversely affected all aspects of development of cell and gene therapies,” said CBER director Peter Marks at the Alliance for Regenerative Medicine’s Meeting on the Mesa. “For some of the studies that are ongoing there are some real challenges to overcome in terms of endpoints that may have been missed.”

Pa­tient death in piv­otal gene ther­a­py tri­al rat­tles Sarep­ta part­ner al­ready fac­ing clin­i­cal hold

A child with mucopolysaccharidosis type IIIA (MPS IIIA) has died in Lysogene’s pivotal trial for its lead gene therapy, four months after the FDA imposed a clinical hold on the study for possible safety concerns.

The study was supposed to pave the path toward commercialization by Sarepta, which inked a $125 million deal in 2018 to market the drug, LYS-SAF302, in the US and worldwide except Europe.

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Eric Kelsic (Dyno)

Roche turns to a Har­vard up­start out of George Church’s lab to con­struct AAV 2.0 mod­el vec­tors — a key part of build­ing the gene ther­a­py pipeline

For a gene therapy tech upstart that launched out of stealth mode just 5 months ago with big goals and not a tremendous amount of launch money, Dyno Therapeutics has quickly moved itself into a frontline offensive in R&D — working in league with some of the marquee developers in the field.

Building on alliances with Novartis and Sarepta, the global Roche/Genentech group has now stepped in with the latest discovery deal, willing to wager up to $1.8 billion that the spinout from George Church’s lab at Harvard has what it takes to deliver on AAV 2.0. There’s an upfront, but the partners are keeping that part mum. But while most early-stage deals are heavily back ended, there’s a big enough number here to make a difference on the bank balance.

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Out to pi­o­neer gene edit­ing in Chi­na, Edi­Gene bags $67M to be­gin clin­i­cal test­ing for β-tha­lassemia ther­a­py, uni­ver­sal CAR-T

A gene editing player hailing from Beijing has clinched $67 million to steer two of its lead candidates into the clinic — and to prove itself as a CRISPR pioneer in China.

EdiGene gathered the Series B from a slate of top-tier Chinese investors, including 3H Health Investment (who led the round), Sequoia Capital China, Alwin Capital and Kunlun Capital. Previous backers, including IDG Capital, Lilly Asia Venture, Huagai Capital and Green Pine Capital Partners, also participated.

Voy­ager shares take a hit as FDA slaps a hold on the IND for Hunt­ing­ton's gene ther­a­py — their #2 play in the pipeline

A little more than a year after Sanofi handed back rights to Voyager’s $VYGR gene therapy program for Huntington’s in exchange for a cash payment, the biotech says the FDA has placed a hold on the IND needed to get into the clinic.

The word from Voyager — the FDA remains mute in these cases — is that the hold on VY-HTT01 deals with CMC issues, something that typically isn’t the kiss of death for a biotech company. But it will put the brakes on things for an uncertain period of time for Voyager, now helmed by Andre Turenne.

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A2 Bio­ther­a­peu­tics nabs $71M-plus Se­ries B for se­lec­tive cell ther­a­py pipeline

More than 15 years after beating cancer himself, A2 Biotherapeutics CEO Scott Foraker has pulled in a $71.5 million Series B round to send a mystery candidate for solid tumors into the clinic.

“It’s quite personal for me and I really am driven to make a difference,” said Foraker, a non-Hodgkin’s lymphoma survivor. “I know what it’s like to sit in an infusion chair for hours and get doses of chemotherapy.”

Io­vance shares ham­mered on TIL ther­a­py fil­ing de­lay — al­though an­a­lysts aren't as both­ered

Iovance won’t be able to file for its first-ever approval by the end of this year after all.

At issue is the potency assays Iovance is using to define what would be the first-ever tumor-infiltrating lymphocyte (TIL) therapy. Regulators want to have more data on the current assays, or potentially see different assays in the BLA, the biotech suggested.

All of that work would push the BLA submission in metastatic melanoma to 2021, when Iovance has completed the required clinical follow-up and finished refining its assays as well as developing alternative ones. That means a launch won’t happen until 2022 at the earliest, with cervical cancer to follow in the same year.

Silviu Itescu, Mesoblast CEO

FDA gives thumbs down for Mesoblast's aGVHD drug, say­ing one sin­gle-arm tri­al is not enough

Mesoblast’s request for a trading halt Thursday turned out to be a negative.

Late Thursday evening, the Australian company announced that the FDA had issued a CRL for remestemcel-L, or Ryoncil, its pediatric acute graft-versus-host disease program. Mesoblast is planning to appeal the decision with a Type A meeting sometime in the next 30 days, and will seek a potential accelerated approval with a post-approval condition for an additional study.

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Silviu Itescu, Mesoblast CEO

Mesoblast asks for trad­ing halt as PDU­FA date slides by. What's up?

Everything was all lined up for Mesoblast’s Wednesday PDUFA date regarding its acute graft-versus-host disease candidate remestemcel-L, or Ryoncil. But with no word in any direction on any FDA actions, the biotech is preparing its stock for the delayed ruling.

Mesoblast $MESO has asked the Australian stock exchange to place its shares in a trading halt until the company can make an announcement about the Ryoncil review. That announcement will likely come on Monday at the latest, the company said in a release.

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Ilan Ganot, Solid Bio

Sol­id gets an­oth­er shot in DMD gene ther­a­py race as FDA lifts hold. But is it too late to catch Pfiz­er, Sarep­ta?

Things can get moving again in Worcester, Massachusetts and Gainesville, Florida, where Solid Biosciences operate their clinical sites.

Nearly a year after the FDA suspended Solid’s Duchenne muscular dystrophy gene therapy trial over safety concerns for a second time, the agency has given the Cambridge biotech the go-ahead to start dosing again. The trial will likely resume in the first quarter of next year, CSO Carl Morris said in a statement.

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