Joel Schneider, Carbon Biosciences president and CEO

Car­bon Bio­sciences emerges with a new vi­ral vec­tor to shake up the cys­tic fi­bro­sis gene ther­a­py field

Car­bon Bio­sciences wants to dis­rupt the field of AAV gene ther­a­py by tap­ping in­to a fam­i­ly of thou­sands of virus­es to find ones that al­low for the “next gen­er­a­tion of vi­ral de­liv­ery ve­hi­cles,” as CEO Joel Schnei­der puts it.

The Mass­a­chu­setts biotech emerged Tues­day with $38 mil­lion in part­ner­ship with the Cys­tic Fi­bro­sis Foun­da­tion to take a bo­cavirus-based — part of the broad­er par­vovirus web — gene ther­a­py in­to the clin­ic for the lung dis­or­der.

Ade­no-as­so­ci­at­ed virus gene ther­a­pies have tripped up the biotech R&D world for years now, with var­i­ous tox­i­c­i­ty is­sues and oth­er lim­it­ing fac­tors, so Car­bon wants to go af­ter virus­es that al­low for gene ther­a­pies to be de­liv­ered more than once, which will open up the op­por­tu­ni­ty to tar­get more pa­tients.

The start­up’s first ther­a­py, named CGT-001, can be de­liv­ered more than once, specif­i­cal­ly tar­gets the lung tis­sue and is able to trans­port the full-length cys­tic fi­bro­sis gene, Schnei­der tells End­points News.

The lead can­di­date is al­so able to car­ry 30% to 40% more than the typ­i­cal AAV, which Schnei­der knows a thing or two about af­ter eight years in the C-suite at AAV-based Sol­id Bio­sciences, which is at­tempt­ing to tack­le Duchenne mus­cu­lar dy­s­tro­phy.

He de­clined to say when Car­bon will en­ter the clin­ic, but not­ed the Se­ries A will get the start­up there. The first hu­man tri­al will tar­get CF pa­tients who don’t have a mod­u­la­tor ther­a­py, Schnei­der said.

Car­bon is the first start­up launched out of the 2020 in­cu­ba­tor cre­at­ed be­tween Long­wood Fund and the Cys­tic Fi­bro­sis Foun­da­tion.

Car­bon is based on sci­ence from John En­gel­hardt, di­rec­tor of Uni­ver­si­ty of Iowa’s Cen­ter for Gene Ther­a­py of Cys­tic Fi­bro­sis, and Robert Kotin, pro­fes­sor of mi­cro­bi­ol­o­gy and phys­i­o­log­i­cal sys­tems at Uni­ver­si­ty of Mass­a­chu­setts Chan Med­ical School.

Lucy Liu

Lucy Liu, VP and co-founder at Car­bon and a se­nior as­so­ciate at Long­wood, re­called call­ing En­gel­hardt at the end of 2020 af­ter the Long­wood-CFF pro­gram launched and at the end of this con­ver­sa­tion, he men­tioned he’d been work­ing on a way to over­come AAV’s lim­i­ta­tions of pre-ex­ist­ing im­mu­ni­ty, small car­go ca­pac­i­ty, lim­it­ed tis­sue tro­pism, etc.

Then, serendip­i­tous­ly with­in a month, Kotin came to Long­wood and CFF say­ing he’d been work­ing on a sim­i­lar idea in his lab. Kotin is now in­ter­im CSO of Car­bon.

So in mid-2021, Car­bon was formed and has been work­ing out of CFF’s labs in Lex­ing­ton, MA. The start­up will open its own lab in Waltham, MA, by year’s end, Schnei­der said.

The sto­ry be­hind the start­up’s name high­lights the bold am­bi­tion Car­bon is on.

“We’re re­al­ly al­low­ing evo­lu­tion and vi­rol­o­gy to take the front seat here, and so car­bon, which is the build­ing block of all life, we’re re­al­ly al­low­ing that to dri­ve our ap­proach where we’re iden­ti­fy­ing al­ready ex­ist­ing virus­es and tak­ing the best part of what they’ve evolved and what they’ve of­fered and turn that in­to a vi­ral vec­tor for us to use for ther­a­peu­tic process­es,” Liu said.

Aside from get­ting in­to that first hu­man study, Car­bon is build­ing out its plat­form by iden­ti­fy­ing oth­er par­vovirus­es to use to tar­get oth­er tis­sues, Schnei­der said. He’s al­so fo­cused on near­ly dou­bling the team from 13 peo­ple to­day to al­most 25 by the end of 2022.

Down the line, the start­up could go be­yond gene ther­a­pies and in­to gene edit­ing.

“Ini­tial­ly, Car­bon Bio will pur­sue its own ther­a­peu­tic de­vel­op­ment, but col­lab­o­ra­tion with an­oth­er Foun­da­tion-fund­ed com­pa­ny, e.g., for the de­liv­ery of a gene-edit­ing ther­a­py is ab­solute­ly part of fu­ture con­sid­er­a­tions,” CFF’s SVP of drug dis­cov­ery, Mar­tin Mense, told End­points in an email.

The Se­ries A was led by Agent Cap­i­tal and in­clud­ed back­ing from Long­wood, Astel­las Ven­ture Man­age­ment, So­las­ta Ven­tures, Uni­ver­si­ty of Tokyo In­no­va­tion Plat­form, Cam­ford Cap­i­tal and the CFF. Adicet Bio pres­i­dent and CEO Chen Schor is board chair.

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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Sen­ate Fi­nance Com­mit­tee lobs more bi­par­ti­san pres­sure on­to PBMs

Congress is honing in on how it wants to overhaul the rules of the road for pharmacy benefit managers, with a Senate Finance Committee hearing Thursday serving as the latest example of the Hill’s readiness to make changes to how pharma middlemen operate.

While pledging to ensure patients and pharmacies “don’t get a raw deal,” Finance Committee Chair Ron Wyden (D-OR) laid out the beginning of what looks like a major bipartisan effort — moves the PBM industry is likely to challenge vigorously.

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Nicklas Westerholm, Egetis Therapeutics CEO

Ac­qui­si­tion talks on­go­ing for Swedish rare dis­ease biotech Egetis, shares up al­most 40%

Shares of the Sweden-based rare disease biotech Egetis Therapeutics skyrocketed on Thursday afternoon as the company said it’s engaged in “ongoing discussion” with external parties regarding a “potential acquisition.”

Egetis confirmed rumors with a statement on Thursday while noting that there is no certainty that a takeover offer will be made.

Nonetheless, the possibility of an acquisition has shot up Egetis’ share price. By the afternoon on Thursday, its stock price was {$EGTX.ST} up over 38%. An Egetis spokesperson told Endpoints News in an email that it has no further comments.

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Lu­pus drug de­vel­op­ment mar­ket heat­ing up, while FDA links with ad­vo­ca­cy group to fur­ther ac­cel­er­ate re­search

The long-underserved systemic lupus erythematosus (SLE) market is suddenly buzzing with treatment possibilities. Less than two years after AstraZeneca’s approval for Saphnelo — the first new SLE drug in a decade and joining just one other approved in GSK’s Benlysta – the pipeline of potential drugs numbers in the dozens.

Although most are very early stage — Spherix Global Insights estimates five in Phase II/III — the pharma R&D enthusiasm is catching on among doctors, patients and advocacy groups. On Wednesday, the Lupus Research Alliance and the FDA formed a novel private-public partnership called Lupus Accelerating Breakthroughs Consortium (Lupus ABC) to help advance lupus clinical trial success.

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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.

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.

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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John Flavin, Portal Innovations CEO

Por­tal In­no­va­tions takes its Chica­go biotech in­vest­ing, lab space mod­el to Boston with Bob Langer start­up in tow

Life sciences investor and lab operator Portal Innovations is taking the blueprint it set in Chicago and expanding to Massachusetts, where it will deploy $25 million, and help house new startups in the former Boston Globe headquarters.

Portal CEO and founder John Flavin told Endpoints News the company will open its Boston-area operations in the coming weeks, and its first lab occupant will be Syntis Bio, an early-stage biotech out of Moderna co-founder Bob Langer, MIT professor Giovanni Traverso and Sherlock Biosciences co-founder Rahul Dhanda.

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