Jude Samulski. AskBio

Plug­ging in the last piece to its gene ther­a­py puz­zle, AskBio ac­quires Scot­tish syn­thet­ic pro­mot­er com­pa­ny

AskBio is putting some of their re­cent $235 mil­lion raise to use, adding an­oth­er key piece to its ex­pan­sive gene ther­a­py toolk­it with the ac­qui­si­tion of Ed­in­burgh, Scot­land-based Syn­promics.

Sheila Mikhail

To be op­er­at­ed as a whol­ly-owned sub­sidiary at arms’-length — in fact, all the way across the At­lantic from AskBio’s home base in Re­search Tri­an­gle Park, NC — Syn­promics brings a gene con­trol syn­thet­ic pro­mot­er tech­nol­o­gy to the ta­ble. These are DNA se­quences that play a cru­cial role in the tran­scrip­tion of genes, specif­i­cal­ly those that drug de­vel­op­ers want to reg­u­late in the con­text of cell and gene ther­a­py.

“For some time, the field has un­der­stood that the three es­sen­tial com­po­nents for ad­vanc­ing suc­cess­ful AAV gene ther­a­py has cen­tered around pro­duc­tion, cap­sids and pro­mot­ers. To­day AskBio has added the last crit­i­cal com­po­nent to our tool chest,” Jude Samul­s­ki, a co-founder and Uni­ver­si­ty of North Car­oli­na Gene Ther­a­py Cen­ter di­rec­tor, said.

Hav­ing sup­plied the vec­tor tech­nol­o­gy that went in­to AveX­is’ pi­o­neer­ing SMA gene ther­a­py (now be­long­ing to No­var­tis) and sold spin­out Bam­boo Ther­a­peu­tics to Pfiz­er, Samul­s­ki and CEO Sheila Mikhail now wants to be­come its own “gen­er­a­tional com­pa­ny” see­ing a pipeline of gene ther­a­pies for rare dis­ease through to the mar­ket. The promise is that their cell line man­u­fac­tur­ing process and ex­ten­sive AAV cap­sid li­brary will prove com­pet­i­tive in a field that’s seen the rise of spe­cial­ized play­ers and at­tract­ed the at­ten­tion of Big Phar­ma BD groups.

The lead prod­ucts, for Pompe and Parkin­son’s re­spec­tive­ly, are in Phase I/II.

Michael Roberts

“In­te­grat­ing our in­ducible pro­mot­er sys­tems in­to the AskBio plat­form will en­able pre­ci­sion con­trol in the next gen­er­a­tion of ther­a­pies,” added Michael Roberts, founder and CSO of Syn­promics, added.

He will re­main in his role along­side CEO David Ven­ables. While fi­nan­cial de­tails were not dis­closed, Cal­cu­lus Cap­i­tal said the re­turn on its £5.6 mil­lion in­vest­ment “sig­nif­i­cant­ly ex­ceeds tar­get re­turns.”

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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Adeno-associated virus-1 illustration; the use of AAVs resurrected the gene therapy field, but companies are now testing the limits of a 20-year-old technology (File photo, Shutterstock)

Af­ter 3 deaths rock the field, gene ther­a­py re­searchers con­tem­plate AAV's fu­ture

Nicole Paulk was scrolling through her phone in bed early one morning in June when an email from a colleague jolted her awake. It was an article: Two patients in an Audentes gene therapy trial had died, grinding the study to a halt.

Paulk, who runs a gene therapy lab at the University of California, San Francisco, had planned to spend the day listening to talks at the American Association for Cancer Research annual meeting, which was taking place that week. Instead, she skipped the conference, canceled every work call on her calendar and began phoning colleagues across academia and industry, trying to figure out what happened and why. All the while, a single name hung in the back of her head.

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Jackie Fouse, Agios CEO

Agios scores its sec­ond pos­i­tive round of da­ta for its lead pipeline drug — but that won't an­swer the stub­born ques­tions that sur­round this pro­gram

Agios $AGIO bet the farm on its PKR activator drug mitapivat when it recently decided to sell off its pioneering cancer drug Tibsovo and go back to being a development-stage company — for what CEO Jackie Fouse hoped would be a short stretch before they got back into commercialization.

On Tuesday evening, the bellwether biotech flashed more positive topline data — this time from a small group of patients in a single-arm study. And the executive team plans to package this with its earlier positive results from a controlled study to make its case for a quick OK.

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Vir's CMO says he's sur­prised that a low dose of their he­pati­tis B drug ap­pears promis­ing in ear­ly slice of da­ta — shares soar

Initial topline data from a Phase I study of a new therapeutic for chronic hepatitis B virus was so promising that it surprised even the CMO of the company that produces it.

Vir Biotechnology on Tuesday announced that its VIR-3434 molecule reduced the level of virus surface antigens present in a blinded patient cohort after eight days of the trial with just a single 6 mg dose. Six of the eight patients in the cohort were given the molecule, and the other two a placebo—all six who received the molecule saw a mean antigen reduction of 1.3 log10 IU/mL, Vir said.

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Eli Lil­ly demon­strates that 2 an­ti­bod­ies beat 1 for guard­ing against se­vere Covid-19. But can that solve the first an­ti­body’s prob­lem amid slow up­take?

It seems safe to say that two antibodies are better than one.

Eli Lilly released the largest results yet on Tuesday for their Covid-19 neutralizing antibody cocktail, announcing that the combo reduced deaths and hospitalizations in coronavirus patients by 70%. Across 1,000 patients, there were 11 such events in the treatment group and 36 in the placebo group.

The breakdown for deaths alone was even starker: 10 in the placebo group and 0 in the treatment group. Lilly added that the drug hit secondary endpoints for reducing viral load and alleviating symptoms, although they did not disclose numbers.

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George Yancopoulos (L) and Len Schleifer (Regeneron)

Re­gen­eron touts pos­i­tive pre­lim­i­nary im­pact of its Covid an­ti­body cock­tail, pre­vent­ing symp­to­matic in­fec­tions in high-risk group

Regeneron flipped its cards on an interim analysis of the data being collected for its Covid-19 antibody cocktail used as a safeguard against exposure to the virus. And the results are distinctly positive.

The big biotech reported Tuesday morning that their casirivimab and imdevimab combo prevented any symptomatic infections from occurring in a group of 186 people exposed to the virus through a family connection, while the placebo arm saw 8 of 223 people experience symptomatic infection. Symptomatic combined with asymptomatic infections occurred in 23 people among the 223 placebo patients compared to 10 of the 186 subjects in the cocktail arm.

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Matt Gline (L) and Vivek Ramaswamy

Scoop: Vivek Ra­maswamy is hand­ing the CEO job to a top lieu­tenant at Roivant — but he’s not ex­act­ly leav­ing the biotech scene

Over the past 7 years since founding Roivant, Vivek Ramaswamy has been a constant blur of biotech building motion.

He launched his first biotech with an Alzheimer’s drug he picked up cheap, and watched the experiment implode in one of the highest profile pivotal disasters seen in the last decade. But it didn’t slow the 30-something exec down; if anything, he hit the accelerator. Ramaswamy blazed global paths and went on to raise billions to spur the creation of a large lineup of little Vants promising big things at a fast pace. He sold off a section of the Vant brigade to Sumitomo Dainippon for $3 billion. And more recently the relentless dealmaker has been building a computational discovery arm to add an AI-driven approach to kicking up new programs and companies, supplementing the in-licensing drive while pursuing advances that have created more than 700 jobs at Roivant, with $2 billion in reserves.

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Ron Cooper, Albireo CEO

Al­bireo just ad­vanced down to the 10-yard line at the FDA. And Ron Coop­er’s team is get­ting prepped for the next big play

When Albireo Pharma’s board $ALBO moved to bring in Ron Cooper as the CEO more than 5 years ago, the development-stage company went with an experienced commercial player who had a big-time position on his resume after running Bristol Myers’ commercial ops in Europe.

Now, after successfully navigating a pivotal study, putting them in a foot race with a rival toward an FDA OK, Cooper is getting a boost from regulators on the last drive back to an arena he understands completely.

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