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.

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

Yong Dai, Frontera Therapeutics CEO

Scoop: Lit­tle-known Or­biMed-backed biotech clos­es $160M round to start gene ther­a­py tri­al

Frontera Therapeutics, a China and US biotech, has closed a $160 million Series B and received regulatory clearance to test its first gene therapy stateside, Endpoints News has learned.

Led by the largest shareholder, OrbiMed, the biotech has secured $195 million total since its September 2019 founding, according to an email reviewed by Endpoints. The lead AAV gene therapy program is for an undisclosed rare eye disease, according to the source.

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AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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DEM BioPharma CEO David Donabedian (L) and executive chair Jan Skvarka

Long­wood sets an­oth­er 'don't eat me' biotech in­to gear with help of for­mer Tril­li­um CEO Jan Skvar­ka

Jonathan Weissman and team are out with a cancer-fighting biotech riding the appetite for those so-called “don’t eat me” and “eat me” signals.

The scientific co-founder — alongside fellow Whitehead Institute colleague Kipp Weiskopf and Stanford biologist Michael Bassik — has launched DEM BioPharma with incubator Longwood Fund and a crop of other investors.

In all, the nascent, 10-employee biotech has $70 million to bankroll hematology- and solid tumor-based programs, including a lead asset that could enter human trials in two to three years, CEO David Donabedian told Endpoints News.

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De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.

Kelly Martin, Radius Health CEO

VC firms take os­teo­poro­sis drug­mak­er Ra­dius Health pri­vate for al­most $900M

After attacks from activist investors and disappointing returns on share prices, Radius Health has now agreed to new ownership, a direction resulting in leaving the Nasdaq.

Radius Health, a biotech out of Massachusetts with one approved product in its arsenal, announced Thursday morning that it agreed to be acquired by two VC firms: Gurnet Point Capital and Patient Square Capital. The deal, worth around $890 million, will include debt assumption and the payout of $1 CVR per share for investors. And on top of that, OrbiMed is providing debt financing.

John Leonard, Intellia CEO

In­tel­li­a's CRISPR pro­gram that ed­its genes di­rect­ly in pa­tients shows dura­bil­i­ty in AT­TR amy­loi­do­sis

The first in vivo CRISPR/Cas9 gene editing program has some new durability data showing sustained reduction of a toxic protein in ATTR amyloidosis at all four dose levels in a small 15-patient study.

Intellia Therapeutics presented the much-anticipated data for its Regeneron-collaborated NTLA-2001 Friday morning, adding to the initial Phase I results it first delivered almost a year ago to the day.