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.”

Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
Improved accessibility to visualisation
Visualisation is a core component of our ability to understand a concept. Ask 10 people to visualise an apple, and each will come up with a slightly different image, some apples smaller than others, some more round, some with bites taken. Acceptable, you say, we can move on to the next part of the story. Now ask 10 people to visualise how HIV’s capsid protein gets arranged into the hexamers and pentamers that form the viral capsid that holds HIV’s genetic material. This request may pose a challenge even to someone with some virology knowledge, and it is that inability to effectively visualise what is going on that holds us back from fully understanding the rest of the story. So how does medical animation help us to overcome this visualisation challenge?

Alexion has racked up a second approval for Ultomiris, the successor therapy to Soliris, as its mainstay blockbuster therapy faces a patent review process that could drastically shorten its patent exclusivity.

The FDA OK for atypical hemolytic uremic syndrome (aHUS) on Friday was widely expected after Alexion posted a full slate of positive Phase III data in January. But regulators also flagged concerns about serious meningococcal infections, slapping a black box warning on the label and mandating a REMS.

Months ago, Foamix leaned on its biggest shareholders — Perceptive Advisors and OrbiMed — to financially grease its wheels, ahead of the FDA decision date for its acne therapy. On Friday, that approval came in — and the topical formulation of the antibiotic minocycline is set for a January launch.

The therapy, Amzeeq (formerly known as FMX101), was approved to treat inflammatory lesions of non-nodular moderate-to-severe acne vulgaris in patients aged 9 and older.

The rising popularity of CD47 has propelled the “don’t-eat-me” signal to household name status in the immuno-oncology world: By blocking that protein, the theory goes, one can stop cancer cells from fooling macrophages. But just as PD-(L)1 merely represents the most fruitful of all checkpoints regulating T cells, Verseau Therapeutics is convinced that CD47 is one of many regulators one can modulate to stir up or tame the immune system.