Jaime Shamonki, Generate CMO

Al­ready sit­ting on a ma­jor stem cell bank, Gen­er­ate looks to grow its re­gen­er­a­tive med­i­cines busi­ness

Gen­er­ate Life Sci­ences is the largest pri­vate new­born stem cell biorepos­i­to­ry, and with the an­nounce­ment of its own in-house man­u­fac­tur­ing fa­cil­i­ty, the com­pa­ny is look­ing to grow its re­gen­er­a­tive med­i­cine pipeline.

Gen­er­ate has es­tab­lished a fa­cil­i­ty in La Jol­la, CA, to pro­vide end-to-end man­u­fac­tur­ing for new­born stem cell bi­o­log­ics. The site will al­low Gen­er­ate to evolve as it adds a per­son­al­ized ther­a­peu­tics de­vel­op­er to its cur­rent busi­ness mod­el. The new site has been de­signed to har­ness mes­enchy­mal stem cells from the tis­sue of the um­bil­i­cal cord.

“With this fa­cil­i­ty, we will have the in-house ca­pa­bil­i­ty to take these stem cells through the com­plete process of col­lec­tion, cryo­genic stor­age and ul­ti­mate­ly bi­o­log­ics man­u­fac­tur­ing,” CMO Jaime Sha­mon­ki said in a press re­lease. “This will al­low us to pow­er our stud­ies in a much more ef­fi­cient and stream­lined man­ner and help us bet­ter serve the fam­i­lies who have stored over 1.2 mil­lion stem cell units with us.”

Sha­mon­ki and her team have the chance to do some­thing spe­cial: They’re work­ing to find a treat­ment for “long Covid,” or the lin­ger­ing side ef­fects from the dis­ease.

But while com­ing up with a long-term so­lu­tion to a prob­lem that’s sure to im­pact peo­ple around the world for years to come is im­por­tant, there’s an added ben­e­fit to the project. The chief med­ical of­fi­cer for Gen­er­ate Life Sci­ences gets to learn how the cells re­pro­gram the im­mune sys­tem, and di­rect those find­ings to­ward dis­cov­er­ing cures for au­toim­mune dis­eases, Ly­me dis­ease and chron­ic fa­tigue syn­drome.

Ear­li­er in June, Gen­er­ate ac­quired Cell Care in­ter­na­tion­al cord blood bank­ing group, with in­cludes Cell Care in Aus­tralia, and In­scep­tion Lifebank and Cells for Life in Cana­da. The move helps Gen­er­ate be­come more glob­al with its reach, and pro­vides ac­cess to fam­i­lies in Cana­da and Aus­tralia.

The com­pa­ny’s reach now stretch­es across three coun­tries, in­clud­ing the US.

Stem cells from um­bil­i­cal cords of­fer an­ti-in­flam­ma­to­ry and tis­sue repar­a­tive prop­er­ties. Gen­er­ate is in the mid­dle of stud­ies right now that are look­ing at tar­get­ing au­toim­mune dis­ease, neu­ro­log­i­cal dis­eases and lung dis­eases. Af­ter back­ing out of the Covid-19 vac­cine field due to over­crowd­ing, Gen­er­ate es­tab­lished a tri­al for pa­tients with res­pi­ra­to­ry com­pli­ca­tions stem­ming from the virus us­ing a ther­a­py de­rived from new­born stem cells. It’s al­so look­ing at clin­i­cal tri­als for a treat­ment for Type 1 di­a­betes.

“We are see­ing this rapid change in our sci­en­tif­ic ori­en­ta­tion of how they can be used, so pre­vi­ous­ly it was trans­plants but sort of low-in­ci­dence con­di­tions for how things could be treat­ed this way, like thank god child­hood leukemia is not all that com­mon,” Sha­mon­ki said. “But now we’re look­ing at re­gen­er­a­tive med­i­cine in­di­ca­tions.”

The com­pa­ny is try­ing to in­vest in both clin­i­cal tri­als with aca­d­e­m­ic part­ners and their own man­u­fac­tur­ing fa­cil­i­ties, to en­able the donor fam­i­lies to eas­i­ly ac­cess their own units, if need­ed for ther­a­pies in the fu­ture. With Gen­er­ate’s ac­qui­si­tion of Cell Care in­ter­na­tion­al, it will al­low for ex­pand­ed ac­cess to do­nat­ed tis­sue and cord blood from a larg­er pool, as well as uti­lize its sci­en­tif­ic and reg­u­la­to­ry knowl­edge spe­cif­ic to each coun­try.

“It’s re­al­ly all about de­liv­er­ing val­ue to the fam­i­lies who’ve banked new­born stem cells with us, as op­posed to try­ing to get 10 bi­o­log­ics to see what we can get through Phase III and what sticks,” Sha­mon­ki said.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

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Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

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Elisabeth Stampa, Medicines for Europe president

As win­ter ap­proach­es, a Eu­ro­pean gener­ics group rais­es alarm over en­er­gy prices for man­u­fac­tur­ers

While colder temperatures are fast approaching, the situation surrounding the rise of energy prices in Europe is hitting businesses of every kind, including generic drug manufacturers.

Medicines for Europe, a group that represents the generic industry on the continent, sent a letter addressed to energy ministers and commissioners concerning inflation and the costs of energy on the supply of generic medicines.

Up­dat­ed: Al­ny­lam re­in­forces APOL­LO-B patisir­an da­ta be­fore head­ing to the FDA

Weeks after uncorking some mostly positive data for patisiran in transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy, Alnylam is bolstering its package with new exploratory and subgroup data before shipping it off to regulators.

The RNAi drug maintained “generally consistent” benefits in efficacy and quality of life across several prespecified subgroups at month 12, Alnylam announced on Friday afternoon, including age, baseline tafamidis use, ATTR amyloidosis type, baseline six-minute walk test score and others.

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Jerome Durso, Intercept Pharmaceuticals CEO

In­ter­cep­t's OCA fails a PhI­II NASH tri­al, rais­ing fresh doubts about its years­long quest for an OK

Intercept Pharmaceuticals has run into another big setback in its yearslong quest to win an approval for OCA in NASH. The biotech put out word Friday morning that its Phase III REVERSE study failed the primary endpoint for the liver disease, sending its share price into a tailspin.

There was no significant improvement in fibrosis among the patients suffering from cirrhosis who were treated with obeticholic acid, with investigators hunting for a minimum 1-stage histological improvement in the disease after 18 months of therapy.

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