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CEO Pavan Cheruvu (Sio Gene Therapies)

'We're not a van­t': Ax­o­vant seeks to for­get the past as the com­pa­ny re­brands to Sio Gene Ther­a­pies

Aiming to shed the legacy of an epic Alzheimer’s fail a few years ago, Axovant $AXGT is changing its name in a full corporate rebrand.

The company announced Tuesday it will now be called Sio Gene Therapies as it completes what’s been a three-year pivot away from Alzheimer’s disease and toward, well, gene therapy. Sio’s new name, pronounced s-EYE-oh, will go into effect Friday, with its new Nasdaq ticker $SIOX replacing its old symbol at market open.

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DSMB hits pause on Voy­ager's Neu­ro­crine-part­nered Parkin­son's gene ther­a­py

Voyager Therapeutics’ clinical plans have hit another snag.

The Cambridge biotech disclosed in their third quarter earnings Monday that the data safety and monitoring board has recommended halting enrollment on their pivotal Phase II trial testing their lead gene therapy for Parkinson’s disease. Voyager and their partner Neurocrine had already paused enrollment because of Covid-19, but will now postpone plans to resume the study.

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Frank Zhang (AP Images)

Leg­end Biotech has a new chair­woman and per­ma­nent CEO. But where is Frank Zhang?

Frank Zhang isn’t going back to Legend Biotech any time soon.

More than a month after the Somerset, NJ-based company disclosed that Zhang was placed under house arrest in China for a customs investigation, it has installed Ying Huang — its CFO and interim CEO since late September — as the permanent leader, while tapping board director Ye (Sally) Wang as the chairwoman.

Zhang used to hold both of those titles. In a statement, Legend said he remains on the board of directors and offered his support through a written proxy. There is no update on his status.

Laurence Reid (Third Rock)

Deci­bel Ther­a­peu­tics rais­es $82M as Lau­rence Reid looks to steer gene ther­a­py piv­ot

Laurence Reid could have picked a better time to take his latest CEO job.

It was January 29 when Decibel Therapeutics announced they were bringing in the former Alnylam and Millennium exec, and the coronavirus was still a distant threat, not even yet named. The job would have been tall enough without a pandemic; after five years and $100 million, Decibel was preparing to pivot its platform in a new direction, working to expand on gene therapy and regeneration. Reid would need to raise the cash to do so.

Doug Ingram, Sarepta CEO (Sarepta Therapeutics)

Cit­ing pan­dem­ic, Sarep­ta de­fers much-an­tic­i­pat­ed DMD gene ther­a­py PhI­II, will head to reg­u­la­tors af­ter 10-per­son study

When Sarepta disclosed in September that the FDA held up their widely-anticipated Phase III trial over an assay, the big question was whether the setback might allow Pfizer to overtake them in the heated race to develop a gene therapy for Duchenne muscular dystrophy.

On Thursday, though, Sarepta’s CEO Doug Ingram ended any speculation on the topic, by announcing the company would defer its Phase III study altogether.

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FDA scut­tles blue­bird's ac­cel­er­at­ed plans for sick­le cell gene ther­a­py, as high­er CMC bar forces 1-year de­lay

In yet another smudge on bluebird bio’s track record for execution, the biotech is pushing back the filing for its gene therapy in sickle cell disease by a year due to a new data requirement from the FDA.

With the setback, bluebird joins a league of gene therapy developers — from BioMarin to Voyager — that have been hampered by CMC issues. While somewhat unexpected, the regulators’ request highlights their stringency around manufacturing and quality control.

Arie Belldegrun speaking at UKBIO19

Pa­tient death mars lat­est cut of da­ta from Al­lo­gene's off-the-shelf CAR-T

Allogene has been riding high since ASCO in May, when early data showed their off-the-shelf CAR-T worked in a handful of patients and that no one rejected their foreign cells. It was a key moment in a field that could remake cell therapy and treatment for certain cancers.

On Wednesday, though, the Arie Belldegrun-founded biotech released results for their multiple myeloma CAR-T and, although the responses were there, so was a red flag: Four patients contracted a serious infection, and one of them died.

Ver­tex, fresh off an R&D miss, flash­es da­ta sug­gest­ing their sick­le cell CRISPR ther­a­py is work­ing

In one of the strongest bits of evidence yet for the effect CRISPR gene editing can have in people with genetic diseases, Vertex and CRISPR Therapeutics showed their experimental therapy kept two sickle patients from experiencing painful incidences and kept patients with another blood disorder from requiring transfusions.

The data, released today in an abstract for the American Society of Hematology, show that five patients with transfusion-dependent beta thalassemia — a disorder where patients produce insufficient hemoglobin — who received the therapy haven’t needed transfusions since the second month after administration. Previously, the patients had received between 23.5 and 61 units of blood per year.

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JW Ther­a­peu­tics hauls in $300M IPO just as liso-cel-in­spired CAR-T nears the fin­ish line in Chi­na

Close to two years after Juno Therapeutics disappeared from Nasdaq, a CAR-T biotech it spawned is settling on a stock exchange on the other side of the globe.

JW Therapeutics, a joint venture between Juno and WuXi AppTec, raised $300 million in its IPO on the Hong Kong Stock Exchange — building on $190 in venture money since launching in 2018.

The pitch is all about bringing cell therapy breakthroughs to China, tapping into American connections that extend into Lyell, a new startup led by Juno co-founder Rick Klausner, and Eureka, a longtime partner of Juno. WuXi, a global CRO, brings expertise in process development and manufacturing to the mix.

Promis­ing mR­NA tech comes with reg­u­la­to­ry, CMC headaches

As the buzz builds around messenger RNA (mRNA) technology’s use for two leading Covid-19 candidates, manufacturers and regulatory professionals are facing facts: This is not simple technology.

Complex manufacturing processes, delivery vehicles that must be treated more as drug substances than excipients, and potential immunogenicity headaches are among the challenges industry faces as this promising technology is harnessed to address an increasing number of health conditions.