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Close­ly-watched in­ter­na­tion­al CRISPR ethics pan­el leaves door ajar for germline edit­ing — one day

In 2017, some of the world’s top scientists and ethicists emerged from over a year of deliberations with a report meant to finally lay down guiding principles for how CRISPR, the awesome-power-awesome-responsibility genome editing tool, should be safely and morally used.

Then, just months later, a scientist named He Jiankui announced he had used the tool to edit embryos and create so-called “CRISPR babies.” That was baffling to the experts who universally preached caution, but so was his next claim: That he had done so while following the — in hindsight, vague — principles set out in the report.

Re­searchers at City of Hope com­bine on­colyt­ic virus with a CAR-T to erad­i­cate sol­id tu­mors in mice

A new combination of existing cancer treatments is showing early signs of potentially cracking open a new approach to treating solid tumors.

In preclinical research published by City of Hope, a California-based non-profit, scientists effectively blended CAR-T therapy with an oncolytic virus to eradicate solid tumors in mice. The virus is genetically engineered to enter the tumors and force them to express the CD19 protein, allowing the CAR-T cells to attack.

Paula Cannon (Credit: Richard Carrasco)

Re­searchers teamed up to de­vel­op a 'three in one' HIV treat­ment — and the NIH is throw­ing in $14.6M

The NIH is pitching $14.6 million into a “three for one” HIV research program led by USC and the Fred Hutchinson Cancer Research Center that aims to strike the need for daily medication — or even achieve a “home run” cure.

The five-year grant will back preclinical studies that combine gene editing with technology to improve bone marrow transplants. The potential therapy would engineer a patient’s own stem cells to fight HIV, and stimulate them to produce new immune cells once reintroduced to the patient.

Sean Nolan, RA Session II

Crossover round? Check. Top team? Check. Taysha sails to an IPO with $100M ini­tial ask

On the same day Novartis erased the AveXis brand from the company, the ex-AveXis crew says their new gene therapy startup is ready for an IPO.

Taysha Gene Therapies has left enough bread crumbs for biotech sleuths to figure out that it’s going down this path. CEO RA Session II, who happily chatted to multiple reporters at launch, declined interviews for the recent Series B (and presumed crossover round); the biotech brought in a gene therapy program developed by its co-founder Steven Gray from Abeona; and days ago it tapped the ex-CMO and ex-CFO of AveXis to join the board, chaired by their former boss Sean Nolan.

Steve Harr, Sana CEO

Game change: A fron­trun­ner in the cell ther­a­py 2.0 field of­fers a first look at their lead ther­a­py. And it’s a doozy

When a pair of CAR-T pioneers from Juno launched Sana Biotechnology back at the beginning of 2019, they had some grand and rather vaguely defined plans on building multiple platforms to engineer cells — part of the whole cell therapy 2.0 movement that’s attracted billions of dollars in financing.

But, like most private startups, CEO Steve Harr and the crew kept the program details quiet, part of the inevitable “stealthy” approach on timelines and treatments you run into with ambitious players.

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Radek Špíšek, Sotio CEO

Sold for pen­nies, Unum gets one last shot at re­demp­tion

Unum Therapeutics’ cell therapy tech is getting one last lease on life after a Czech biotech was able to pick up the core of a once-ballyhooed company for the cost of spare parts.

For $8.1 million upfront and just $3.4 million in milestones, Prague-based cell therapy startup Sotio bought out the preclinical “BOXR” CAR-T platform that Unum said they would turn to in March, after a third round of safety issues in as many years proved the final straw for their lead program.

Paul Dabrowski, Synthego CEO (Noam Galai/Getty Images for TechCrunch)

Syn­thego adds $100M from RA, Welling­ton, 8VC in pur­suit of 'vir­tu­al­iz­ing bi­ol­o­gy'

The Peter Thiel-backed, Jennifer Doudna-advised, SpaceX engineer-founded CRISPR platform company just added a new mega-round.

Synthego announced $100 million in Series D funding led by Wellington Management, RA Capital Management and 8VC. The company’s second $100 million-plus round since 2018, the new cash will help Synthego advance its platform of CRISPR assays, screens and engineered cell lines.

De­railed gene ther­a­py study re­ports 3rd death as safe­ty and dura­bil­i­ty is­sues cloud a boom­ing field

A third patient has died in Audentes’ pivotal gene therapy trial for X-linked myotubular myopathy, adding fresh concerns for the field right on the heels of BioMarin’s surprise slapdown at the FDA.

The biotech, bought out by Astellas just 8 months ago for $3 billion, says the third patient died from gastrointestinal bleeding. And like the two others who died, this person was on the high dose.

According to Audentes:

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Barbara Weber, Tango Therapeutics CEO

Daniel O’Day’s I/O dance con­tin­ues, as Gilead and Tan­go ex­pand deal up to $6B

Three years ago, Gilead CEO John Milligan signed a milestone-heavy deal with a little-known biotech that had flashy technology and flashier founders. Now, with new chief Daniel O’Day on a cancer therapy shopping spree, the two companies are upgrading that deal into a potential behemoth.

Gilead and Boston’s Tango Therapeutics agreed to an up to $6 billion deal that will cover 15 different targets that help cancer cells evade the immune system, paving the way for drugs that could be used in combination with checkpoint inhibitors for the rest of their growing immuno-oncology portfolio.

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Taysha buys clin­ic-ready gene ther­a­py from Abeona, bring­ing Steven Gray's port­fo­lio back in one place

Less than four months after Taysha launched with a suite of AAV gene therapies developed at UT Southwestern — and two weeks after the ex-AveXis team raised $95 million in fresh cash — the biotech is looking to its chief scientific advisor for another asset.

The new program is ABO-202, which is designed to treat CLN1 disease (or infantile Batten disease) by delivering a correct copy of the PPT1 gene with a modified AAV9 vector.