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Michael Higgins, Voyager interim CEO

Trou­bled Voy­ager’s gene ther­a­py re­launch gets a boost with $630M Pfiz­er deal

It was spring, the pandemic seemed in retreat, patrons were filling Boston’s bars again and, in Cambridge, Voyager Therapeutics was in trouble.

The Third Rock-founded biotech emerged in 2014 as one of the first of the flashy new gene therapy companies, promising one-time treatments for common devastating conditions like Alzheimer’s. It was one of the first to deliver results, too: By 2017, its Parkinson’s treatment had improved motor function in 10 patients.

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Henrietta Lacks (Via Johns Hopkins Medicine)

Es­tate of Hen­ri­et­ta Lacks sues Ther­mo Fish­er over the im­prop­er sale of her im­mor­tal cells

The family of a black woman whose immortal cells were unethically taken from her at Johns Hopkins hospital in 1951 is now suing Thermo Fisher Scientific for profiting from the cells for decades.

The world-famous cell line, known for Henrietta Lacks as HeLa and the subject of a bestselling book about a decade ago, have proven to be incredibly important in human history, helping scientists to make major strides, from the polio vaccine to in vitro fertilization.

Carsten Brunn, Selecta Biosciences CEO

Has Se­lec­ta found a way around gene ther­a­py's de­liv­ery prob­lem? Take­da puts down $1B-plus to find out

While gene therapies were traditionally thought of as one-and-done treatments, scientists now see a benefit in redosing patients down the road. The only problem? Some patients mount an immune response to the adeno-associated viruses (AAV) used to deliver the therapies.

It’s one of gene therapy’s most pressing issues, and a raft of companies are working on non-viral delivery methods to get around it. But Selecta Biosciences thinks it’s found another way to tamp down the body’s immune response — and Takeda is ready to dole out more than $1 billion to get in on the action.

Jim Mullen, Editas Medicine CEO

Ed­i­tas' CRISPR-based eye ther­a­py proves safe, but the ju­ry's still out on whether it works

Researchers at Editas Medicine revealed long-anticipated data for their CRISPR gene editing program Wednesday morning, the second-ever batch of human results for in vivo administration of the Nobel Prize-winning technology. And while the therapy appeared remarkably safe, a huge step forward in its own right, experts questioned whether the early efficacy figures will ultimately bear fruit.

Editas’ data come from a Phase I/II study evaluating a gene editing candidate for LCA10, a form of inherited blindness, with the first cut looking at six adult patients across two low- and mid-dose cohorts. There were no dose-limiting toxicities and most adverse events stemmed from the surgical procedure used to inject the treatment into the eye, Editas said. It was safe enough for the independent data monitoring committee to rubber stamp testing the experimental drug in children.

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Safe­ty fears force Pfiz­er to change piv­otal DMD gene ther­a­py tri­al pro­to­col

As one of the biggest players in an increasingly packed gene therapy space, Pfizer has taken an early lead over specialists like Sarepta in taking a Duchenne muscular dystrophy (DMD) candidate into late-stage testing. But new safety fears have led Pfizer to scale back that trial, cutting out patients with certain genetic mutations.

Pfizer has amended its enrollment protocol for a Phase III test for gene therapy fordadistrogene movaparvovec in DMD after investigators flagged severe side effects tied to specific mutations, according to a letter the drugmaker sent to Parent Project Muscular Dystrophy, a patient advocacy group.

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Mi­cro­cap gene ther­a­py play­er says three chil­dren suf­fered se­ri­ous side ef­fects in PhI/II study

A small biotech looking to develop a gene therapy for a rare condition that can cause color or legal blindness ran into some significant safety trouble in a Phase I/II dose-escalation study.

Applied Genetic Technologies Corporation, also known as AGTC, reported Thursday afternoon that three of five children in the study’s highest dose cohort experienced severe inflammation one month after dosing. The events were concerning enough to be categorized as suspected unexpected serious adverse reactions, or SUSARs.

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UNC re­searchers re­port a new way to charge CAR-T against sol­id tu­mors

When researchers first started putting CAR-T cells into cancer patients in the 1990s, the cells didn’t do very much. They swam around in the blood, killing very little of what they were meant to kill, before dying a quiet death some months later.

There were several big reasons for the failures. Researchers at first picked the wrong diseases, going after solid tumors that, it turned out, were uniquely adept at keeping out T cells. And they didn’t even really know which parts of these tumors were the best to aim at.

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Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a specific market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

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Dave Lennon, former president of Novartis Gene Therapies

Zol­gens­ma patent spat brews be­tween No­var­tis and Re­genxbio as top No­var­tis gene ther­a­py ex­ec de­parts

Regenxbio, a small licensor of gene therapy viral vectors spun out from the University of Pennsylvania, is now finding itself in the middle of some major league patent fights.

In addition to a patent suit with Sarepta Therapeutics from last September, Novartis is now trying to push its smaller partner out of the way. The Swiss biopharma licensed Regenxbio’s AAV9 vector for its $2.1 million spinal muscular atrophy therapy Zolgensma.

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The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

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