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.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Spe­cial re­port 2022: Meet 20 women blaz­ing trails in bio­phar­ma R&D

When you run a special report for a fourth year, it can start feeling a little bit like a ritual. You go through the motions — in our case opening up nominations for top women in biopharma R&D and reviewing more than 500 entries — you make your choices of inclusion and exclusion. You host a ceremony.

But then things happen that remind you why you do it in the first place. Perhaps a Supreme Court rules to overturn the constitutional right to abortion and a group of women biotech leaders makes it clear they strongly dissent; perhaps new data on gender diversity in the industry come out that look all too similar to the old ones, suggesting women are still dramatically underrepresented at the top; perhaps protests and conflicts around the world put in stark terms the struggles that many women still face in earning the most basic recognition.

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Graphic: Shutterstock

Prometheus moves to raise cash hours af­ter PhII da­ta leads to stock surge

After releasing better-than-anticipated data on two mid-stage studies Wednesday morning, Prometheus Biosciences’ CEO said the company would “take some time to assess” its next financing options.

It only needed about seven hours. Wednesday afternoon after the market closed, the biotech announced it would seek $250 million through an equity offering as the company looks to edge out anti-TL1A competitor Pfizer and its new partner Roivant.

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Kristen Hege, Bristol Myers Squibb SVP, early clinical development, oncology/hematology and cell therapy (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Q&A: Bris­tol My­er­s' Kris­ten Hege on cell ther­a­py, can­cer pa­tients and men­tor­ing the next gen­er­a­tion

Kristen Hege leads Bristol Myers Squibb’s early oncology discovery program carrying on from the same work at Celgene, which was acquired by BMS in 2019. She’s known for her early work in CAR-T, having pioneered the first CAR-T cell trial for solid tumors more than 25 years ago.

However, the eminent physician-scientist is more than just a drug developer mastermind. She’s also a practicing physician, mother to two young women, an avid backpacker and intersecting all those interests — a champion of young women and people of color in STEM and life sciences.

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Gossamer Bio CEO Faheem Hasnain at Endpoints' #BIO22 panel (J.T. MacMillan Photography for Endpoints News)

Gos­samer’s Fa­heem Has­nain de­fends a round of pos­i­tive PAH da­ta as a clear win. But can these PhII re­sults stand up to scruti­ny?

Gossamer Bio $GOSS posted a statistically significant improvement for its primary endpoint in the key Phase II TORREY trial for lead drug seralutinib on Tuesday morning. But CEO Faheem Hasnain has some explaining to do on the important secondary of the crucial six-minute walk distance test — which will be the primary endpoint in Phase III — as the data on both endpoints fell short of expectations, missing one analyst’s bar on even modest success.

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Alex Martinez, Intrinsic Medicine CEO

They with­drew their IPO. Then, they broke off their SPAC merg­er. Now what?

If at first an IPO doesn’t succeed, try, try a SPAC. But what happens when that fails too?

Intrinsic Medicine and its blank-check partner Phoenix Biotech Acquisition Corp. called off their reverse merger Tuesday night, citing “current market conditions” as the reason it went kaput. The pair decoupled just weeks after agreeing to combine in late October as investors’ appetite for new IPOs and SPACs has been limited, at best.

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Jay Lichter, Arialys Therapeutics CEO (Avalon Ventures)

Scoop: Aval­on, MPM back new CNS biotech with sci­en­tif­ic chops from Astel­las

A preclinical central nervous system biotech is in the works in La Jolla, CA, and the drug developer has reeled in capital from a syndicate of investors, Endpoints News has learned.

Arialys Therapeutics filed incorporation documents in the Golden State last December and applied its name for trademark protection with the US Patent and Trademark Office the week prior to that. Paperwork with the SEC also outlines plans to offer up equity in exchange for $55 million.

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Piper Trelstad, head of CMC, Bill & Melinda Gates Medical Research Institute

Q&A with Gates leader: Women tak­ing on more roles in phar­ma man­u­fac­tur­ing, but still work to do

More and more women are driving innovation and taking leadership roles in biotech – as evidenced today in the release of Endpoints News’ list of the top 20 women in the R&D world – but those gains are beginning to extend across pharma sectors.

In pharma manufacturing in the US today, around 46% of all roles are occupied by women, according to the US Bureau of Labor Statistics for 2021. And according to a Bloomberg report, women’s roles across manufacturing roles had a massive boost after the start of the pandemic.

Phar­ma rep­u­ta­tion re­tains 'halo' even as pan­dem­ic me­dia cov­er­age re­cedes — sur­vey

The Covid-19 halo effect on the pharma industry is continuing, according to a new global study from Ipsos. The annual survey for the International Federation of Pharmaceutical Manufacturers and Associations (IFPMA) finds considerable goodwill from consumers across measures of trust, cooperation with governments, and advancing research and drug development.

“Despite the pandemic in many countries no longer being the top of mind concern generally – although it does remain the top concern as a health issue – the industry’s reputation has remained positive,” said Ipsos research director Thomas Fife-Schaw.

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