Vec­talys and Flash­Cell merge in­to Flash Ther­a­peu­tics, loaded with RNA pipeline and man­u­fac­tur­ing set-up

Pas­cale Bouil­lé (La Tri­bune Toulouse / Bor­deaux, YouTube)

Two gene ther­a­py com­pa­nies in France are merg­ing to cre­ate one ven­ture called Flash Ther­a­peu­tics, com­bin­ing man­u­fac­tur­ing know-how with a pipeline of RNA drug prospects.

The merg­er is be­tween Vec­talys, a Toulouse-based mak­er of lentivi­ral vec­tors, and its spin­out start­up Flash­Cell, which launched out of Vec­talys just last year to tack­le RNA car­ri­ers for drug ap­pli­ca­tions.

The CEO of the com­bined com­pa­ny, Pas­cale Bouil­lé, tells me the merg­er part­ly came down to bio­man­u­fac­tur­ing in­dus­tri­al log­ic.

“Man­u­fac­tur­ing ca­pa­bil­i­ty is crit­i­cal to the suc­cess of this new field, so it makes sense to in­te­grate man­u­fac­tur­ing with the ther­a­peu­tics busi­ness and to lever­age our lentivi­ral de­vel­op­ment and man­u­fac­tur­ing ex­per­tise with oth­er in­dus­try play­ers,” Bouil­lé said.

Flash will be tasked with ad­vanc­ing nov­el RNA drugs based on the com­pa­ny’s Lenti­Flash tech­nol­o­gy, a pro­pri­etary lentivi­ral de­liv­ery method. Lentivirus­es, a sub­class of retro­virus­es, are used as vec­tors for gene ther­a­py thanks to their abil­i­ty to in­te­grate in­to the genome of non-di­vid­ing cells. That’s a unique fea­ture, as oth­er retro­virus­es can in­fect on­ly di­vid­ing cells.

Bouil­lé tells me cur­rent lentivi­ral vec­tors are a lead­ing de­liv­ery method for CAR-T and oth­er ge­net­ic dis­eases that re­quire sta­ble, long-term ex­pres­sion. But some next-gen im­munother­a­py (for ex­am­ple, those in­volv­ing cell types be­yond T cells) may need tran­sient ex­pres­sion rather than in­te­gra­tive, long-term ex­pres­sion. En­ter Lenti­Flash, which is “non-in­te­gra­tive,” al­low­ing tran­sient and short-term RNA ex­pres­sion.

Flash Ther­a­peu­tics will take on the dis­cov­ery and de­vel­op­ment of “stand­alone pro­grams in the ar­eas of liv­er and blood dis­ease” that were based on Lenti­Flash and cul­ti­vat­ed at Flash­Cell over the past year.

The com­pa­ny’s lentivi­ral de­vel­op­ment and pro­duc­tion busi­ness, which will con­tin­ue to op­er­ate un­der the Vec­talys man­u­fac­tur­ing plat­form name, will ex­pand as part of Flash Ther­a­peu­tics to in­clude GMP man­u­fac­tur­ing ca­pa­bil­i­ties. The GMP fa­cil­i­ty, which is ex­pect­ed to be­come ful­ly op­er­a­tional in 2019, is be­ing es­tab­lished through a re­cent­ly signed, three-year part­ner­ship with Hos­pi­tal Saint-Louis, Lari­boisière, Fer­nand Vi­dal to de­vel­op and pro­duce gene and cell ther­a­py drugs.

ZS Per­spec­tive: 3 Pre­dic­tions on the Fu­ture of Cell & Gene Ther­a­pies

The field of cell and gene therapies (C&GTs) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C&GTs that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.

Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.

Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.

Graphic: Kathy Wong for Endpoints News

What kind of biotech start­up wins a $3B syn­di­cate, woos a gallery of mar­quee sci­en­tists and re­cruits GSK's Hal Bar­ron as CEO in a stun­ner? Let Rick Klaus­ner ex­plain

It started with a question about a lifetime’s dream on a walk with tech investor Yuri Milner.

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Milner’s question on one of those mornings on foot: “What do you want to do?”

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Sec­ondary patents prove to be key in biosim­i­lar block­ing strate­gies, re­searchers find

While the US biosimilars industry has generally been a disappointment since its inception, with FDA approving 33 biosimilars since 2015, just a fraction of those have immediately followed their approvals with launches. And more than a handful of biosimilars for two of the biggest blockbusters of all time — AbbVie’s Humira and Amgen’s Enbrel — remain approved by FDA but still have not launched because of legal settlements.

Hal Barron (GSK via YouTube)

GSK R&D chief Hal Bar­ron jumps ship to run a $3B biotech start­up, Tony Wood tapped to re­place him

In a stunning switch, GlaxoSmithKline put out word early Wednesday that R&D chief Hal Barron is exiting the company after 4 years — a relatively brief run for the man chosen by CEO Emma Walmsley in late 2017 to turn around the slow-footed pharma giant.

Barron is being replaced by Tony Wood, a close associate of Barron’s who’s taking one of the top jobs in Big Pharma R&D. He’ll be closer to home, though, for GSK. Barron has been running a UK and Philadelphia-based research organization from his perch in San Francisco.

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Chamath Palihapitiya and Pablo Legorreta

Bil­lion­aires Chamath Pal­i­hapi­tiya and Pablo Legor­re­ta hatch an $825M SPAC for cell ther­a­py biotech

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ProKidney was founded 6 years ago but really got going at the beginning of 2019 with the $62 million acquisition of inRegen, which was working on an autologous — from the patient — cell therapy for kidney disease. After extracting kidney cells from patients, researchers expand the cells in the lab and then inject them back into patients, aiming to restore the kidneys of patients suffering from CKD.

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CBO: Medicare ne­go­ti­a­tions will ham­per drug de­vel­op­ment more than pre­vi­ous­ly thought

As President Biden’s Build Back Better Act — and, with it, potentially the Democrats’ last shot at major drug pricing reforms in the foreseeable future — remains on life support, the Congressional Budget Office isn’t helping their case.

The CBO last week released a new slide deck, outlining an update to its model on how Medicare negotiations might take a bite out of new drugs making it to market. The new model estimates a 10% long-term reduction in the number of new drugs, whereas a previous CBO report from August estimated that 8% fewer new drugs will enter the market over 30 years.

Joshua Brumm, Dyne Therapeutics CEO

FDA or­ders DMD tri­al halt, rais­ing ques­tions about a whole class of promis­ing drugs

Dyne Therapeutics’ stock took a nasty hit this morning after the biotech put out word that the FDA had slapped a clinical hold on their top program for Duchenne muscular dystrophy. And now speculation is bouncing around Biotwitter that there could be a class effect at work here that would implicate other drug developers in the freeze.

Dyne execs didn’t have a whole lot to say about why the FDA sidelined their IND for DYNE-251 in DMD while “requesting additional clinical and non-clinical information for” the drug.

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UCB buys its way to epilep­sy show­down with Jazz with $1.9B Zo­genix ac­qui­si­tion

Zogenix’s epilepsy drug Fintepla may only have brought in around $100 million of sales in its first year, but UCB clearly believes it can go much, much higher.

The Belgian pharma has inked a $1.9 billion deal to buy out Zogenix, paying $26 per share in cash and offering a contingent value right worth $2 more per share if Fintepla lands an extra EU approval by the end of 2023.

But even the upfront marks a 72% premium to California-based Zogenix’s shares, which were trading just north of $15 on Tuesday.

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