Dave Greenwald (Deerfield)

Deer­field-backed biotech emerges from stealth with gene ther­a­py de­liv­ery plat­forms from Har­vard and MIT

As a PhD stu­dent at Tufts, Dave Green­wald stud­ied the use of ade­no-as­so­ci­at­ed virus (AAV) vec­tors to de­liv­er gene ther­a­pies to peo­ple with re­tini­tis pig­men­tosa, a group of rare ge­net­ic eye dis­eases that caus­es the reti­na to break down over time.

While he lat­er left lab life, he now sits at the helm of Aper­tu­ra Gene Ther­a­py, a biotech that hopes to cus­tomize AAV vec­tors that are used wide­ly in de­liv­er­ing most gene ther­a­pies. Aper­tu­ra an­nounced its launch from stealth with $67 mil­lion in Se­ries A fi­nanc­ing from Deer­field, where Green­wald is al­so a vice pres­i­dent of busi­ness de­vel­op­ment.

Ben De­v­er­man

The com­pa­ny ac­quired its AAV tech­nol­o­gy from Ben De­v­er­man, a Broad In­sti­tute sci­en­tist who has spent near­ly 15 years study­ing AAV vec­tors.

“When de­vel­op­ing a gene ther­a­py, it has been com­mon to use nat­u­ral­ly oc­cur­ring serotype AAV cap­sids,” De­v­er­man said in a press re­lease.

On the oth­er hand, his lab’s tech­nol­o­gy de­signs “cus­tom AAV cap­sids that have the cho­sen char­ac­ter­is­tics for treat­ing spe­cif­ic dis­eases,” he said, “and we be­lieve this ap­proach will re­sult in new and ef­fec­tive gene ther­a­pies.”

When asked which spe­cif­ic dis­eases Aper­tu­ra plans on go­ing af­ter with the cus­tom AAV cap­sids, Green­wald said, “The nice thing about these plat­forms is that they are ag­nos­tic to cell type and or­gan, so we ac­tu­al­ly use them in pret­ty much any dis­ease.”

How­ev­er, he told End­points News, “we’re not dis­clos­ing spe­cif­ic ther­a­peu­tic ar­eas or in­di­ca­tions at this time.”

Kristi­na Wang

Giv­ing slight­ly more in­sight, Aper­tu­ra’s di­rec­tor of cor­po­rate de­vel­op­ment Kristi­na Wang said, “Nat­ur­al serotype AAVs don’t reach cer­tain tis­sues very well, such as CNS, kid­ney, and a bunch of oth­er tis­sues. We are en­gi­neer­ing AAVs to get to those dif­fi­cult-to-ac­cess tis­sues.”

Aper­tu­ra al­so plans to in­cor­po­rate an­oth­er gene ther­a­py tech­nol­o­gy, known as PESCA, that it ac­quired from a dif­fer­ent Cam­bridge-area sci­en­tist — Michael Green­berg, the chair of Har­vard’s neu­ro­bi­ol­o­gy de­part­ment.

While the AAV tech­nol­o­gy is about what goes on around the cap­sid, “the tech­nol­o­gy from Har­vard is in­side the cap­sid, so it’s more about the reg­u­la­tion,” Green­wald said. “So once we get the gene ther­a­py to the right place, do we turn it on in the right cells at the right amount?”

PESCA can fine-tune the ge­net­ic pay­load of a ther­a­py, “po­ten­tial­ly avoid­ing off-tar­get tox­i­c­i­ties, and com­bined with our AAV cap­sids, can po­ten­tial­ly avoid some of the high sys­temic dosage re­quire­ments as in some of the cur­rent gene ther­a­pies,” added Wang, who worked in Green­berg’s lab while she at­tend­ed grad school at Har­vard.

The tox­i­c­i­ty from high dos­es of AAV-de­liv­ered gene ther­a­pies has been an on­go­ing safe­ty con­cern. In a re­port pub­lished last year, FDA reg­u­la­tors not­ed that 35% of near­ly 150 tri­als with AAV gene ther­a­pies re­port­ed se­ri­ous ad­verse events, in­clud­ing deaths, re­lat­ed to the AAV-de­liv­ered treat­ments.

Aper­tu­ra hopes to present its pre­clin­i­cal da­ta lat­er this year, Green­wald said.

Bio­mark­er 'roadmap­s' and the fu­ture of can­cer R&D; Cur­tain rais­es on #AS­CO22; Pfiz­er, No­var­tis tack­le drug ac­cess; 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.

While this was not a week for earth-shattering news, there were certainly a lot of interesting tidbits. If you found this recap helpful, please recommend it to your friends and colleagues. We’ll see you on the other side of the long weekend.

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Keep­ing pres­sure on Am­gen, Mi­rati draws mixed re­views on lat­est cut of KRAS da­ta

As the close runner-up to Amgen’s Lumakras in the KRAS race, any data cut from Mirati’s adagrasib continues to draw scrutiny from analysts. And the latest batch of numbers from ASCO is a decidedly mixed bag.

While a quick comparison suggests that adagrasib spurred slightly more responses and led to a longer overall survival than Lumakras among a group of non-small cell lung cancer patients, its duration of response appears shorter and the safety profile continues to spark concern.

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Ann is one of ViiV Healthcare's newest spokespeople as the retired school administrator speaks up about her HIV status.

GSK's Vi­iV de­buts next evo­lu­tion in HIV med Dova­to cam­paign with new spokes­peo­ple and new mes­sage

When Ann saw the first TV commercials for HIV medicine Dovato, she didn’t see herself represented. So the 74-year-old retired school administrator who’s been living with HIV since 1998, reached out to GSK’s ViiV Healthcare and asked why not?

Now Ann is one of three people starring in ViiV’s latest Dovato campaign called “Detect This.” The next-step evolution in the branded campaign plays on the word “detect” — often used in describing HIV status under control as undetectable — but in this case, uses the word as a directive for people to understand they can use fewer medicines.

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Tran­si­tion to new Eu­ro­pean clin­i­cal tri­als in­fo sys­tem starts slow­ly

At the end of January, the European Medicines Agency officially launched its new clinical trials info system (CTIS), although the migration to the new platform has only really just begun, and sponsors have until the end of January 2023 before all initial trial applications must be submitted through CTIS.

Overall, 56 clinical trial applications have been submitted in CTIS during the first 3 months since the launch of the system on Jan. 31, according to new data posted by the EMA. By comparison, about 4,000 new trials are authorized each year across Europe.

Switzer­land to de­stroy over 600,000 ex­pired dos­es of Mod­er­na Covid vac­cine

As concerns related to uptake and distribution continue to linger, Switzerland is among the first countries that plans to destroy hundreds of thousands of expired and unused Covid-19 vaccine doses.

The European country said it plans to destroy more than 600,000 doses of Moderna’s Spikevax Covid-19 vaccine as the doses have reached their expiration date.

However, Moderna CEO Stéphane Bancel told the World Economic Forum in Davos, Switzerland that he’s in the process of throwing 30 million doses in the garbage, exclaiming, “We have a big demand problem.”

Lanny Sun, co-founder, CEO and chairman of Full-Life Technologies

Se­quoia Chi­na leads $37M in­fu­sion in­to ra­dio­phar­ma­ceu­ti­cals play­er set­ting up shop in Chi­na and Bel­gium

It’s not just American startups that are tuning into the rising interest in radiopharmaceuticals.

Sequoia China is leading a $37 million Series A into Full-Life Technologies, a biotech headquartered in Shanghai with offices in Brussels, Belgium, to develop a pipeline of radioactive cancer therapies.

The idea isn’t new: As clinicians started routinely deploying radiation to kill cancer cells, scientists and drugmakers have long been exploring ways to limit that powerful effect only to cancer cells while sparing healthy cells. But recent progress in the production of radioisotopes — coupled with big investments from Big Pharma, most notably Novartis and Bayer — has inspired a new wave of startups.

Nassim Usman, Catalyst Biosciences CEO

Af­ter $60M Ver­tex deal, group of Cat­a­lyst share­hold­ers claims biotech could’ve sold as­sets three years ago

Catalyst Biosciences was down to five employees in March, and the biotech needed to do something after two rounds of layoffs, a nixed collaboration and a culling of its hemophilia program.

In came Vertex, with $60 million to buy up the South San Francisco biotech’s preclinical complement drugs, which target the system that bridges the body’s innate and adaptive immune response and a class most known for Ultomiris and Soliris. The deal includes CB 2782-PEG, the dry AMD drug that Biogen no longer wanted in March.

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Lina Khan, FTC chair (Graeme Jennings/Pool via AP Images)

Pile-on over PBMs con­tin­ues with FTC com­ments and a new bi­par­ti­san Sen­ate bill

More than 500 stakeholders sent comments to the FTC on whether the commission should look further into pharma middlemen, known as PBMs, with many of the commenters calling for more federal oversight.

Similar to the critical open comment period in a deadlocked FTC session last February, pharmacies and pharmacy groups are continuing to call out the lack of transparency among the top 3 PBMs, which control about 80% of the market.

Pharma brands are losing their shine with US consumers who are now thinking about the economy and inflation instead of Covid. (Credit: Shutterstock)

Phar­ma brands fade in an­nu­al Har­ris con­sumer vis­i­bil­i­ty poll: Mod­er­na drops off and Pfiz­er dips

As Covid-19 concerns are fading in the US, so is biopharma visibility. The annual Axios Harris Poll survey to determine and rank the 100 most top-of-mind brands in the US finds Moderna, which was No. 3 last year, not on the list at all for 2022, and Pfizer sinking 37 spots.

However, it’s not that Moderna or Pfizer did anything wrong, it’s just that Americans have moved on to other worries beyond Covid.

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