Eye­ing a trio of tri­al ini­ti­a­tions, Jim Wilson's gene ther­a­py start­up woos Bruce Gold­smith from Deer­field as CEO

Pas­sage Bio — Jim Wil­son’s self-de­scribed “lega­cy com­pa­ny” — has wooed a sea­soned biotech ex­ec­u­tive to steer the clin­i­cal en­try of its first three gene ther­a­py pro­grams.

Bruce Gold­smith

Bruce Gold­smith jumps to the helm of Pas­sage af­ter a brief CEO stint at Civet­ta, a can­cer-fo­cused start­up he helped launch while a ven­ture part­ner at Deer­field. He takes over from Or­biMed part­ner and in­ter­im chief Stephen Squin­to, who will now lead the R&D team.

He joins as the biotech preps IND fil­ings for three lead pro­grams in rare, mono­genic dis­eases of the cen­tral ner­vous sys­tem in 2020 — the lyso­so­mal stor­age dis­or­ders GM1 gan­gliasi­do­sis and Krabbe dis­ease, as well as fron­totem­po­ral de­men­tia.

“Bruce is ide­al­ly suit­ed to lead Pas­sage Bio as chief ex­ec­u­tive of­fi­cer giv­en his strong neu­ro­science back­ground cou­pled with his ro­bust health­care and biotech­nol­o­gy in­dus­try ex­pe­ri­ence,” board chair­man Tachi Ya­ma­da said in a state­ment.

Pas­sage launched last Feb­ru­ary with $115 mil­lion from a mar­quee group of Se­ries A in­vestors in­clud­ing Fra­zier (where Ya­ma­da is a part­ner), Or­biMed, Ver­sant Ven­tures, New Leaf Ven­ture Part­ners, Vi­vo Cap­i­tal and Lil­ly Asia Ven­tures. With an of­fice just a 10-minute walk away from Wil­son’s lab at the Uni­ver­si­ty of Penn­syl­va­nia, the com­pa­ny was de­signed to ap­ply the gene ther­a­py pi­o­neer’s 35-year ex­pe­ri­ence in­to “cross-cor­rec­tion­al ther­a­pies” for CNS.

Stephen Squin­to

Ac­cord­ing to what he calls the Jim Wil­son 90/10 rule, Squin­to pre­vi­ous­ly told End­points News, AAV vec­tors can cov­er and trans­duce 90% of mo­tor neu­ron cells but on­ly 10% to 15% of oth­er brain cells — mak­ing it dif­fi­cult to go af­ter in­di­ca­tions where broad trans­duc­tion is need­ed. But it can still prove use­ful in dis­or­ders that re­sult from mu­ta­tions in en­zymes that can be tak­en up by neigh­bor­ing cells once se­cret­ed nor­mal­ly.

A close pact with Penn’s Gene Ther­a­py Pro­gram and Or­phan Dis­ease Cen­ter gave Pas­sage rights to five pro­grams right out of the gate, with op­tions to li­cense sev­en more.

“It’s a very ag­gres­sive clin­i­cal de­vel­op­ment strat­e­gy across a mul­ti­tude of pro­grams,” Squin­to said as he closed a $110 mil­lion Se­ries B in Sep­tem­ber. “We’re not gonna re­ly on any one pro­gram to dri­ve the val­ue of Pas­sage, we’re gonna re­ly on what is a very very full pipeline of op­por­tu­ni­ties.”

Gold­smith will now lead a team of about 25 to build on pre­clin­i­cal and IND-en­abling da­ta from Wil­son’s lab — a com­pa­ny grow­ing ex­er­cise he honed as COO of Lyc­era. There, he was al­so cred­it­ed for a num­ber of busi­ness de­vel­op­ment ini­tia­tives.

The tran­si­tion in­to the clin­ic would al­so mean mov­ing pro­duc­tion from ear­ly fa­cil­i­ties at Penn to Paragon’s GMP sites, and even­tu­al­ly to a cus­tomized suite slat­ed for com­ple­tion in the third quar­ter of this year.

Squin­to, a rare dis­ease ex­pert who de­vot­ed much of his ca­reer to Alex­ion, will con­tin­ue to help over­see all of that as a board di­rec­tor.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

The End­points 11: They've got mad mon­ey and huge am­bi­tions. It's time to go big or go home

These days, selecting a group of private biotechs for the Endpoints 11 spotlight begins with a sprint to get ahead of IPOs and the M&A teams at Big Pharma. I’ve had a couple of faceplants earlier this year, watching some of the biotechs on my short list choose a quick leap onto Nasdaq or into the arms of a buyer.

Vividion, you would have been a great pick for the Endpoints 11. I’m sorry I missed you.

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Dave Lennon, former president of Novartis Gene Therapies

So what hap­pened with No­var­tis Gene Ther­a­pies? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

Emma Walmsley, GlaxoSmithKline CEO (Credit: Fang Zhe/Xinhua/Alamy Live News)

The fire un­der Glax­o­SmithK­line's Em­ma Walm­s­ley grows as an­oth­er well-known ac­tivist in­vestor grabs its pitch­fork — re­port

Bluebell Capital Partners, a proxy brawler fresh off a campaign to oust global food giant Danone’s CEO and most of its board of directors, has bought a stake in UK drugmaker GlaxoSmithKline with its eyes trained directly on Emma Walmsley, the Financial Times reported Wednesday.

The London-based hedge fund joins another notorious activist firm in Paul Singer’s Elliott Management, which earlier this year called for a shakeup in leadership at GSK to handle what the company described as a wealth of riches across the drug giant’s portfolio hindered by limited vision from top staff.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

FDA au­tho­rizes Pfiz­er's vac­cine boost­er for se­niors, those at high risk for se­vere Covid-19

The Biden administration’s goal of kicking off its booster shot drive for the entire US population this week is not quite going as planned.

First, Pfizer applied for approval of a supplemental application for the booster shots, but since last Friday’s adcomm reviewing them, the plan has devolved into an EUA, which the FDA issued late Thursday evening.

The population that is now eligible for the booster, six months after receiving the first pair of Pfizer-BioNTech vaccines, also narrowed from what Pfizer applied for (everyone who’s eligible for the initial Pfizer shots) to just those who are 65 or older, or at high-risk of a Covid infection, including health care workers and others with occupational hazards.

Stéphane Bancel, AP Images

Fi­nal analy­sis of US-fund­ed Mod­er­na Covid vac­cine tri­al shows 98% ef­fi­ca­cy against se­vere dis­ease

A final look at the results of the placebo-controlled Moderna trial in the New England Journal of Medicine, published Thursday afternoon, shows how the vaccine continues to prevent Covid-19 and severe cases after more than five months following the second shot.

Of the more than 30,000 enrolled in the trial that ultimately led to the vaccine’s EUA, only two people in the vaccine group got a severe form of the disease, compared to 106 in the placebo group — leading to an efficacy of 98%.

FDA+ roundup: Bs­U­FA III ready for show­time, court tells FDA to re-work com­pound­ing plan, new guid­ance up­dates and more

The FDA has now spelled out what exactly will be included in the third iteration of Biosimilar User Fee Act (BsUFA) from 2023 through 2027, which similarly to the prescription drug deal, sets fees that industry has to pay for submitting applications, in exchange for firm timelines that the agency must meet.

This latest deal includes several sweeteners for the biosimilar industry, which has yet to make great strides in the US market, with shorter review timelines for safety labeling updates and updates to add or remove an indication that does not contain efficacy data.

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