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.

Paul Hudson, Sanofi CEO (Cyril Marcilhacy/Bloomberg via Getty Images)

FDA side­lines Paul Hud­son's $3.7B MS drug af­ter es­tab­lish­ing link to liv­er dam­age

One of Sanofi CEO Paul Hudson’s top picks in the pipeline — picked up in a $3.7 billion buyout 2 years ago — has just been sidelined in the US by a safety issue.

The pharma giant put out word early Thursday that the FDA has put their Phase III studies of tolebrutinib in multiple sclerosis and myasthenia gravis on partial clinical hold, halting enrollment and suspending dosing for patients who have been on the drug for less than 60 days. Patients who have completed at least 60 days of treatment can continue therapy as researchers explore a “limited” — but unspecified in Sanofi’s statement — number of cases of liver injury.

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Phar­ma re­acts to post-Roe; Drug­mak­ers beef up cy­ber de­fense; Boehringer, Roche qui­et­ly axe drugs; 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.

As a reminder, we are off on Monday for the Fourth of July. I hope this recap will kick off your (long) weekend well and that the rest of it will be just what you need. See you next week for a shortened edition!

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Eric Hughes, incoming Teva EVP of global R&D and CMO

Te­va chief raids Ver­tex for his new glob­al head of re­search and de­vel­op­ment

Teva CEO Kåre Schultz has found his new R&D chief and CMO in Vertex’s ranks.

The global generics giant, which has some 3,500 staffers in the R&D group, has named Eric Hughes to the top research spot in the company. He’ll be replacing Hafrun Fridriksdottir, who held the role for close to five years, on Aug. 1.

Hughes hasn’t been at Vertex for long, though. He jumped from Novartis less than a year ago, after heading the immunology, hepatology & dermatology global development unit. Before that, he completed a five-year stint as head of early clinical research for the specialty discovery medicine department in the exploratory clinical & translational research group at Bristol Myers Squibb, according to his LinkedIn profile.

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#BIO22: Man­ag­ing a biotech in tur­bu­lent times. 'There's a per­fect shit­show out there'

On Tuesday, June 14, Endpoints News EIC John Carroll sat down with a group of biotech execs to discuss the bear market for industry stocks and how they were dealing with it. Here’s the conversation, which has been lightly edited for brevity.

Martin Meeson, sponsor opening:

Thank you, John. Hello everyone. My name’s Martin Meeson, I’m the CEO of Fujifilm Diosynth. For those of you who don’t know Fujifilm Diosynth, we operate in the development of clinical and commercial product scale up, we have facilities in Europe and the US, and around about 4,000 employees. We run on average about 150 programs, so when it comes to managing in turbulent times over the last two years, we’ve had quite a lot of experience of that. Not just keeping the clinical pipelines and the commercial pipelines open, but also our response to the pandemic and the molecules that we’ve had within there. One of the phrases that I coined probably about a year ago when we were talking at JP Morgan, was I talked about managing through turbulent times. Well, it’s become the fact that we are not managing and leading through these times, we are managing in them, which is why that’s really the purpose of and the topic that we’ve got today.

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On Friday, Lonza announced plans to construct a large-scale commercial drug product fill and finish facility in the town of Stein, Switzerland.

Lon­za to in­vest $500M+ on fill-fin­ish fa­cil­i­ty on its home turf

Lonza has been expanding its reach across the globe, bringing sites in China and the US online this year, but now they are looking closer to home for their next major investment.

The Swiss manufacturer on Friday announced plans to construct a large-scale commercial drug fill and finish facility in the town of Stein, Switzerland. The new facility will be delivered through an investment of approximately CHF 500 million, or $519 million, and is expected to be completed in 2026. The facility will also be constructed on the same campus as Lonza’s current clinical drug product facility.

Bo Cumbo, AavantiBio CEO

Scoop: A small gene ther­a­py biotech, flush with ex-Sarep­ta and blue­bird lead­ers, guts CMC

En route to entering the clinic with its first AAV-based gene therapy for a rare neuromuscular disease, AavantiBio has let go of 30 employees, Endpoints News has learned.

The move comes after a year stacking its executive bench with ex-Sarepta and bluebird bio leaders and inking multiple partnerships with the likes of Aldevron, Catalent and Resilience. The biotech also formed a scientific advisory board in February.

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Kate Haviland, Blueprint Medicines CEO

What bear mar­ket? Blue­print lines up $1.25B to ex­pand la­bels, maybe tack on more drugs

As it works to pad the case for expanding its Ayvakit and Gavreto labels, Blueprint Medicines has lined up $1.25 billion in funding, with some of that money seemingly earmarked for acquisitions or pipeline expansion projects.

Following the likes of BioCryst, Cytokinetics and MorphoSys, Blueprint is aiming to monetize the royalties of its RET+ non-small cell lung cancer and thyroid cancer drug Gavreto with Royalty Pharma. The investment group will dole out $175 million upfront and might tack on another $165 million in biobucks as part of Blueprint’s royalties receivable from net sales of the drug by Roche outside the US, sans China.

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Fu­ji­film in­vests an­oth­er $1.6B in­to its CD­MO arm to up­grade facil­lites in the US and Eu­rope

Fujifilm’s spending spree into its CDMO arm is not slowing down.

The multinational announced on Wednesday that it will invest $1.6 billion to enhance and expand the cell culture manufacturing services of the CDMO arm of the Japanese conglomerate Fujifilm Diosynth.

The investment will enhance Fujifilm Diosynth Biotechnologies’ sites in Hillerød, Denmark, and College Station, TX. The investment is expected to create approximately 450 jobs across both facilities.

Anand Parikh, Faeth Therapeutics CEO

Sid Mukher­jee, Lew Cant­ley be­hind new can­cer biotech with food+drug com­bo treat­ment am­bi­tions 

Famed oncologist Sid Mukherjee and repeat biotech co-founder Lew Cantley have teamed up to form Faeth Therapeutics, a startup aiming to treat cancer the way other conditions are addressed: pairing nutrition with therapeutics.

The goal is to transform cancer treatment with nutrition and make it the fourth “pillar” in the oncology regimen, which to date has centered on radiotherapy, surgery and drugs, Faeth CEO Anand Parikh told Endpoints News. Other conditions have already been addressed with a side of nutrients or diet, he said, pointing to diabetes, irritable bowel syndrome and other diseases.

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