Shelia Violette (Q32)

For a post-Soliris world, At­las-backed Q32 Bio out­lines $46M next-gen com­ple­ment play

Long be­fore Alex­ion kin­dled a re­nais­sance of com­ple­ment ther­a­peu­tics with the in­tro­duc­tion of the first an­ti-C5 an­ti­body, Mike Holers — a long­time pro­fes­sor at the Uni­ver­si­ty of Col­orado School of Med­i­cine — be­came fas­ci­nat­ed with the host de­fense sys­tem as he com­plet­ed his rheuma­tol­ogy train­ing. As Soliris be­gins to fade and fol­low-on, ri­vals and even gener­ics catch up to the stan­dard bear­er. Holers is de­but­ing a next-gen­er­a­tion ap­proach he’s been re­fin­ing with At­las Ven­ture over the last two years.

Fund­ed with $46 mil­lion pro­vid­ed by At­las as well as Or­biMed, Abing­worth and Sanofi Ven­tures, Q32 wants to bring the first tis­sue-tar­get­ed com­ple­ment drugs in­to the clin­ic by the end of 2021. The CU Health­care In­no­va­tion Fund and Chil­dren’s Hos­pi­tal Col­orado are al­so in­vestors.

Michael Holers

Over his years of re­search and col­lab­o­ra­tion with col­league Joshua Thur­man and Steven Tom­lin­son at the Med­ical Uni­ver­si­ty of South Car­oli­na, Holers told End­points News, the com­ple­ment field has evolved to re­veal tar­gets that can help sci­en­tists ze­ro in on dis­eased tis­sue, rather than block­ing com­ple­ment ac­ti­va­tion sys­tem­i­cal­ly.

When you think through how the com­ple­ment sys­tem func­tions, you see “a lot of high lev­els of sys­temic pro­teins, but its re­al tis­sue in­jury, its re­al dam­age is lo­cal­ized to cells and tis­sues on the sur­face of cells, in the in­ter­sti­tium,” he said.

Yet know­ing how to di­rect drugs to the right places wasn’t enough, as his ex­pe­ri­ence with a pre­vi­ous start­up dubbed Tal­i­gen — which had an ear­ly tis­sue tar­get­ing tech­nol­o­gy that was ac­quired but ul­ti­mate­ly re­tired by Alex­ion — showed. You al­so need deep knowl­edge in pro­tein en­gi­neer­ing to make sure you’re rein­ing in the com­ple­ment sys­tem prop­er­ly.

That’s where At­las and She­lia Vi­o­lette, Q32’s co-founder, CSO and pres­i­dent of re­search, comes in.

Rather than an­ti­bod­ies, they are work­ing on fu­sion pro­teins that grab the tis­sue-spe­cif­ic tar­gets on one end and car­ries a nat­u­ral­ly oc­cur­ring pro­tein that nor­mal­ly keeps the sys­tem in check.

“One of the things that is now bet­ter un­der­stood is that across pret­ty much any dis­ease where com­ple­ment ac­ti­va­tion be­comes dys­reg­u­lat­ed, it’s be­cause you’re los­ing the op­ti­mal func­tion of those neg­a­tive reg­u­la­tor pro­teins,” Vi­o­lette, who left a lengthy ca­reer at Bio­gen to be­come At­las’ en­tre­pre­neur-in-res­i­dence in 2016, said.

It’s so cen­tral to what they do that the start­up re­named it­self from Ad­mirx to Q32, draw­ing in­spi­ra­tion from the re­gion of the chro­mo­some where these mol­e­cules re­side, she added; the num­ber 32 al­so is a shoutout to the im­por­tance of strat­e­gy, em­bod­ied by chess.

Michael Brox­son

The lead can­di­date blocks both C3 and C5 con­ver­tases, Holers said, play­ing in­to mul­ti­ple parts of the sys­tem. Their ini­tial fo­cus will be in kid­ney and skin dis­eases.

On top of the plat­form play, Q32 al­so in-li­censed an un­re­lat­ed an­ti­body that blocks IL-7 re­cep­tors from Bris­tol My­ers Squibb. The deal closed in Oc­to­ber, as Bris­tol My­ers Squibb was auc­tion­ing off as­sets on the back of its Cel­gene buy­out. At that point, it was al­so close to nom­i­nat­ing its first de­vel­op­ment can­di­date on the com­ple­ment side.

“In the spir­it of full trans­paren­cy,” CEO Mike Brox­son, a Take­da vet and for­mer chief of Goldfinch Bio who of­fi­cial­ly came on board in Feb­ru­ary, said, “but for Covid we might have launched the com­pa­ny sev­er­al months ago.”

While the com­pound, ADX-914, re­sides in the realm of adap­tive im­mu­ni­ty rather than in­nate im­mu­ni­ty where its tech plat­form op­er­ates, it pur­sues a sim­i­lar strat­e­gy not to com­plete­ly knock out a prob­lem­at­ic path­way but fine tune it. (Just don’t ex­pect any more such deals; af­ter this the 12-per­son team will fo­cus en­tire­ly on the com­ple­ment plat­form.)

“We’re re­al­ly about restor­ing home­osta­sis,” Brox­son said.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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Stéphane Bancel, Moderna CEO (AP Photo/Markus Schreiber)

Mod­er­na so­lid­i­fies deal with Kenya to build mR­NA man­u­fac­tur­ing fa­cil­i­ty

The mRNA player Moderna is further cementing its presence on the African continent.

Moderna announced on Thursday that it has finalized an agreement with Kenya’s government to partner up and bring an mRNA manufacturing facility to the east African nation. The new facility aims to manufacture up to 500 million doses of vaccines annually. Moderna also said the new facility will have the ability to spike its production capabilities to respond to public health emergencies on the continent or globally.

Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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Man­u­fac­tur­ing roundup: Catal­ent to pro­duce low-cost ver­sion of nalox­one; CSL opens R&D site

Catalent will be manufacturing a low-cost version of the opioid overdose treatment naloxone as part of a contract with Harm Reduction Therapeutics.

Catalent plans to manufacture the treatment at its facility in Morrisville, NC. No financial details on the deal were disclosed.

Harm Reduction was granted priority review status for the NDA on its spray last year. The company has been working on a naloxone product since 2017. It is anticipating approval in July of this year and a US launch in early 2024.

As­pen looks to re­bound in pro­duc­tion and rev­enue af­ter Covid-19

Last year, South African-based vaccine manufacturer Aspen Pharmacare was facing reports that it had not received a single order for its manufactured Covid-19 shots and that manufacturing lines were sitting idle. But now the vaccine producer is looking to turn things around.

Aspen’s disclosure of its financial results in March unveiled that manufacturing revenue had decreased by 12% to R 603 million ($33.8 million), which Lorraine Hill, Aspen Group’s COO, said is attributable to lower Covid vaccine sales.

Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

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Ribbon cutting ceremony for Thermo Fisher's new cell therapy manufacturing site in San Francisco

Ther­mo Fish­er moves on cam­pus with new cell man­u­fac­tur­ing site in San Fran­cis­co

Thermo Fisher Scientific is putting down more roots in the Bay Area.

The manufacturer opened the doors to a new cell therapy manufacturing facility next to the University of California-San Francisco Medical Center’s Mission Bay campus and on the university’s campus.

UCSF and Thermo Fisher have had a partnership since 2021, with the new site focusing on manufacturing cell therapeutics for certain cancers, including glioblastoma and multiple myeloma. The new site plans to use Thermo Fisher’s expertise in manufacturing services to help UCSF accelerate the development of cell therapies and eventually get them into the clinic, said Dan Herring, the general manager of cell therapy services at Thermo Fisher, in an interview with Endpoints News.

Sulagna Bhattacharya, Nanoscope Therapeutics CEO

Nanoscope’s eye dis­ease gene ther­a­py shows mixed re­sults in PhII

Dallas-based biotech Nanoscope Therapeutics unveiled Phase II results on its gene therapy for a rare eye disease Thursday morning.

In the RESTORE trial, 18 patients with retinitis pigmentosa got a gene therapy called MCO-010 while nine got placebo. On a vision test called the MLYMT, the treatment group had a one-point greater change over one year in their score compared to the placebo group, the primary endpoint of the study. However, the 95% confidence interval was 0.0 to 3.0, meaning the result was not statistically significant. The p-value was not provided.