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.”

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Back in November, the Department of Health and Human Services took the rare step of filing a complaint against Gilead for infringing on government-owned patents related to the HIV drug Truvada (emtricitabine/tenofovir disoproxil fumarate) for pre-exposure prophylaxis (PrEP).

But on Thursday, Gilead filed its own retort, making clear that it does not believe it has infringed on the Centers for Disease Control and Prevention’s (CDC) Truvada patents because they are invalid.

→ Less than a year ago Gilead’s antiviral remdesivir failed to make the cut as investigators considered a raft of potential drugs that could be used against an Ebola outbreak. But it may gain a new mission with the outbreak of the coronavirus in China, which is popping up now around the world.

Gilead put out a statement saying that they’re now in discussions with health officials in the US and China about testing their NUC against the virus. It’s the latest in a growing lineup of biopharma companies that are marshaling R&D forces to see if they can come up with a vaccine or therapy to blunt the spread of the virus, which has now sickened hundreds, killed at least 17 people and led the Chinese government to start quarantining cities.

Back at the beginning of 2015, Deerfield Management co-led a $10 million Series C for a private gene therapy startup, reshaping the company and bringing in new leaders to pave way for an IPO just a year later.

Fast forward four more years and the startup, AveXis, is now a subsidiary of Novartis marketing the second-ever gene therapy to be approved in the US.

For its part, Deerfield has also grown more comfortable and ambitious about the nascent field. And the investment firm is now putting down its biggest bet yet: a $1.1 billion contract development and manufacturing facility to produce everything one needs for cell and gene therapy — faster and better than how it’s currently done.

Different therapeutic traits brandished by the three approved therapies for sickle cell disease all extend life expectancy, but their impact on quality of life is uncertain and their long-term cost-effectiveness is not up to scratch according to the thresholds considered reasonable by ICER, the non-profit concluded in a draft guidance report on Thursday.

Sickle cell disease (SCD), which encompasses a group of inherited red blood cell disorders that typically afflict those of African ancestry, impacts hemoglobin — and is characterized by episodes of searing pain as well as organ damage.

Six years after Kura Oncology snagged a farnesyl transferase inhibitor from J&J and leapt straight into clinical development, one of the biotech’s founders is leaving to start a new chapter in his career.

CMO and development chief Antonio Gualberto is exiting the company, and Kura — led by longtime biotech entrepreneur Troy Wilson — is on the hunt for a replacement. Wilson credited the CMO for some key biomarker work, including the discovery of the CXCL12 pathway as a target of their lead drug tipifarnib. Those biomarkers are being relied on to define the patient population most likely to benefit from the drug.