Cell/Gene Tx channel feed

The long-over­looked safe­ty is­sue weigh­ing on the FDA’s mind on day 1 of marathon gene ther­a­py meet­ing

As the FDA closed out day 1 of its marathon gene therapy hearing, the agency gave an unusual glimpse into their conversations — and conflict — with companies trying to start gene therapy trials.

Routinely, FDA official Andrew Byrnes explained in the only followup question regulators asked during the 8-hour session, the agency has been forced to grapple with an impurity known as empty capsids — a long known but little discussed potential safety issue for gene therapies — and, crucially, how much to push back on companies who refuse to remove them because of how costly or time consuming it might be.

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James Li, JW Therapeutics CEO

Sis­ter to Juno's liso-cel, JW's rel­ma-cel lands sec­ond-ever CAR-T ap­proval in Chi­na

Juno may have stumbled its way to a distant third finish in the CD19 CAR-T race, but its joint venture in China is heading off to a thriving start.

JW Therapeutics, which took its name from co-founders Juno and WuXi AppTec, has scored the second-ever CAR-T approval by China’s National Medical Product Administration just weeks after Fosun Kite, another joint venture, claimed first.

Now sanctioned to treat relapsed or refractory large B-cell lymphoma patients who’ve had two or more lines of systemic therapy, relma-cel was developed on the same cell process platform that spawned Juno’s liso-cel — now Bristol Myers Squibb’s Breyanzi — but a different drug in that it’s tailored for Chinese needs.

FDA posed a sprawl­ing set of ques­tions for two-day gene ther­a­py hear­ing. Here's what com­pa­nies and ex­perts will be watch­ing for

Gene therapy is having a moment. Once confined to academic labs and a couple of companies, the field now encompasses hundreds of trials, dozens of companies and billions of investment dollars.

With greater power, though, comes greater responsibility. The FDA is asking a panel of outside experts to try and define those responsibilities as they debate a lengthy list of 15 questions Thursday and Friday, in a two-day marathon meeting on the future of a field that has promised one-time treatments for some of the most famous and fatal diseases known to medicine.

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UP­DAT­ED: Astel­las paus­es Au­den­tes' gene ther­a­py pro­gram again af­ter 'ab­nor­mal' liv­er tests in yet an­oth­er set­back

Audentes’ troubled the gene therapy program for a rare and devastating muscle wasting disease isn’t yet out of the woods.

The Astellas subsidiary has once again halted a trial for its X-linked myotubular myopathy candidate, the Japanese Pharma announced, after a serious adverse event emerged in which researchers observed “abnormal liver function tests” within the first month of dosing. The hold is voluntary for now, but Astellas left open the possibility of receiving an official FDA notice.

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Eric Ostertag, Poseida CEO (via website)

Mov­ing past a pa­tient death, Po­sei­da of­fers an ear­ly snap­shot of proof of ef­fi­ca­cy for a next-gen sol­id tu­mor CAR-T

Ten months after the FDA took the shackles off Poseida’s study for a CAR-T directed against castrate-resistant prostate cancer, the executive team is now rolling out an early snapshot of the proof-of-concept data it’s looking for in a small, early-stage study.

Tested in 9 heavily pre-treated patients — with an average of 6 prior therapies — investigators tracked a significant, 50%-plus drop in PSA levels in 3 of those patients and what they called a “concordant” imaging result for tumor effect. One patient had a complete response — the ideal result for any cancer study.

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Feng Zhang (Susan Walsh/AP Images)

Feng Zhang's lab de­vel­ops po­ten­tial break­through in RNA edit­ing de­liv­ery us­ing 'ul­tra­com­pact' ver­sions of Cas13

As one of the pioneers behind CRISPR, the Broad Institute’s Feng Zhang has fashioned himself into a gene editing Renaissance man in recent years with a finger in every pie. For RNA editing, one of his passion projects, Zhang has worked to crack a chronic delivery logjam — and his team has just made a big breakthrough there.

A team of researchers out of Zhang’s lab has built a pair of “ultracompact” RNA editing tools that can fit inside an adeno-associated virus (AAV) package, potentially creating a breakthrough in the delivery of those gene editing tools into human cells, according to an article published in Nature Biotechnology on Monday.

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Laronde CEO Diego Miralles and Flagship general partner Avak Kahvejian

Big mon­ey: Flag­ship's uber-plat­form play Laronde rakes in $440M to chase pro­gram­ma­ble 'eR­NA'

With the success of Moderna, parent company Flagship Pioneering has become the mothership for even more audacious next-gen platform plays. Investors like what they see, and now they’ve opened the vault to a particularly bold Flagship startup working on ring-shaped RNA it thinks could be the future of drugmaking.

Laronde has closed a $440 million Series B — one of the largest ever fundraising rounds of its type — to pursue a programmable RNA platform the biotech has previously said could churn out 100 marketed drugs or drug programs in 10 years.

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Ap­peals court sides with Gilead, Kite in CAR-T patent brawl, void­ing Bris­tol My­ers’ big $1.2B award

A new twist emerged Thursday in the long-running patent fight between Juno Therapeutics and Gilead’s Kite Pharma over CAR-T technology, one that gets Kite off the hook for more than $1.2 billion in damages.

The US Court of Appeals for the Federal Circuit reversed a previous decision awarding the money to Juno, ruling in a 19-page opinion that a patent owned by the Bristol Myers Squibb subsidiary and Sloan Kettering is invalid. All three panel judges agreed with Kite’s position, overturning a verdict from December 2019 after finding “substantial evidence does not support the jury’s verdict in Juno’s favor.”

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Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

No­var­tis' CAR-T fails to help a vul­ner­a­ble group of NHL pa­tients — quash­ing hope of mov­ing up lines of treat­ment

Novartis’ efforts to move its CAR-T into earlier lines of treatment have hit a brick wall.

Kymriah, its pioneering CD19-targeting therapy, has failed to bend the curve on event-free survival for patients with second-line aggressive B-cell non-Hodgkin lymphoma when compared with standard of care. While the treatment arm was given infusions of engineered T cells, the SOC group had salvage chemotherapy followed — in responding patients — by high-dose chemotherapy and stem cell transplant.

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Cap­ping AAV dose? Tweak­ing gene ther­a­py tri­als? New an­i­mal mod­els? FDA pos­es far-reach­ing ques­tions for ex­perts

Is it time to set a limit for how high a dose gene therapy developers and investigators are allowed to give patients in each trial?

That’s one of the questions the FDA is posing to its Cellular, Tissue, and Gene Therapies Advisory Committee as experts prepare for a two-day meeting next week. Depending on how the discussion goes — and what the agency makes of it — the results could completely redefine the rules for a major section of the booming gene therapy field: treatments that are delivered by adeno-associated virus vectors.