Cell/Gene Tx channel feed

What were End­points read­ers tun­ing in­to this year? Here’s a look at our 15 most pop­u­lar re­ports of the year (so far)

At the beginning of this year, I laid out a basic objective for Endpoints News as we headed to our 5th anniversary. We’ve long been doing a fine job covering the breaking news in R&D — if I do say so myself — but we needed to expand our horizons on industry coverage, increase the staff and go much, much deeper when the stories demanded it.

In a phrase: broader and deeper.

It’s safe to say, based on our daily web traffic, that you all seemed to like this idea. We’ve doubled the staff — thanks to a growing group of paid subscribers — ramped up the daily report and now publish a regular slate of in-depth articles. And traffic — those clicks you always read about — have gone up in volume too. Monthly sessions are up 43%, to close to 1.5 million. Unique readers are up 63%, to 874,480 in October, after setting a record of close to a million the month before. Page views are running at 3 million-plus a month. And the overall number of subscribers has surged to 124,000.

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Ronald Lorijn, NeuExcell CEO

NeuEx­cell heads east, land­ing sub­ur­ban Philly head­quar­ters

Pennsylvania’s already well-established biotech scene got word of a boost last week with several announcements, including the building of a massive life sciences manufacturing center in Pittsburgh thanks to some funding from an area nonprofit and a local university. But those jawns in Philadelphia aren’t letting go of its stranglehold on the state easily.

NeuExcell Therapeutics, a preclinical gene therapy biotech that focuses on neurodegenerative diseases, announced that it signed a deal with The Discovery Labs in King of Prussia, about 25 miles northwest of downtown Philly.

Affimed CEO Adi Hoess

Af­fimed spurs promis­ing re­sponse num­bers in NK cell study, but dura­bil­i­ty will be the big ques­tion

German biotech Affimed caught some attention earlier this year with a pair of complete responses in an early study of its NK cell regimen for lymphoma. The biotech is back with more data from that study, and the results look promising — but will durability hold up?

A combination regimen of donor NK cells and Affimed’s CD30-targeting innate cell engager AFM13 spurred responses in 16 of 18 patients with relapsed or refractory Hodgkin and non-Hodgkin lymphomas, including seven complete responses, as part of the first of two rounds of treatment in a Phase I/II study, the biotech said Monday.

Blue­bird bio may fi­nal­ly get a gene ther­a­py ap­proved in the US

Bluebird bio may, at long last, get a gene therapy approved in the US.

The Cambridge, MA biotech announced Monday that the FDA had accepted and given priority review for Zynteglo, its gene therapy for the rare blood disorder beta-thalassemia. The announcement sets up an expedited FDA decision by May 20, 2022.

A priority review doesn’t guarantee approval, and more than a few recent biotechs have been spurned after receiving the designation, including Incyte, Sesen, and Provention. But it represents a substantial step forward for a therapy that has seen repeated setbacks and a company that not long ago had an application slapped down with a refuse-to-file letter.

Stem cell spe­cial­ist Ncar­dia bags new funds to help scale man­u­fac­tur­ing ops

A Belgian cell therapy company has secured $60 million in funding to strengthen its manufacturing operations, and grow the cell and gene therapy ecosystem across the world.

Ncardia landed the funding from Kiniciti, a US investment platform backed by Welsh, Carson, Anderson & Stowe, a New York City private equity company. The move gives Kiniciti a controlling stake in Ncardia to enhance anything from discovery to clinical programs to commercialized production, the company said in a press release.

WuXi of­fi­cial­ly opens cell and gene ther­a­py site in Philly; An­oth­er mas­sive man­u­fac­tur­ing site comes to NC

A new cell and gene therapy testing facility in Philadelphia’s Navy Yard is officially opened, WuXi ATU announced Monday.

The new facility includes 140,000 square feet worth of laboratories, and will enhance the company’s contract testing, development and manufacturing organization business model by tripling the company’s previous capacity.

The move helps strengthen the existing testing capacity and capability, and combines the company’s powerful testing capabilities with its advanced therapies’ process development and manufacturing platforms, such as TESSA technology for AAV manufacturing and XLenti stable solutions for lentiviral manufacturing, it says in a press release.

Terry Rosen, Arcus CEO

Gilead goes all in on its Ar­cus al­liance, pay­ing $725M cash for rights to a slate of can­cer drugs. And there's no room for fail­ure

Gilead is going all in — hook, line and sinker — on its oncology alliance with Arcus. And they are going for broke.

The big biotech unveiled a deal that now delivers $725 million in opt-in payments covering the clinical development programs for Arcus, ranging from their closely watched anti-TIGIT programs for domvanalimab and AB308 to etrumadenant (the A2a/A2b adenosine receptor antagonist) and quemliclustat, the small molecule CD73 inhibitor. Gilead will also cover half of the development costs, handing Terry Rosen’s biotech a deal that gives them a clear cash runway to achieving all its goals in oncology.

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FDA+ roundup: ALS bill would pro­vide $100M an­nu­al­ly to ac­cess po­ten­tial drugs; FDA needs to bet­ter en­force Clin­i­cal­Tri­als.gov re­port­ing, re­searchers say

The House Energy & Commerce Committee began marking up a dozen bills on Wednesday morning including one that would require the FDA to craft a five-year action plan for fostering the development of drugs that improve or extend the lives of people living with rare neurodegenerative diseases.

Rare neurodegenerative diseases, like amyotrophic lateral sclerosis or ALS, have been historically very difficult to treat. But this bipartisan bill, introduced by Rep. Mike Quigley (D-IL), will provide $100 million for each of fiscal years 2022 through 2026 to help HHS award grants to facilitate access to investigational drugs that diagnose or treat ALS.

Catherine Stehman-Breen and Vic Myer, Chroma CEO and CSO

At­las, New­path throw $125M be­hind a new ap­proach to gene edit­ing from the field’s biggest names

A handful of the world’s most prominent gene editing-focused academics have been working for over a year on a new company built around a new approach for modifying DNA to treat disease. Known as Chroma Medicine, it launched on Wednesday with $125 million in early funding from Atlas, Newpath, Cormorant and several other VCs.

Chroma will focus on a markedly different way of modifying the genome than most of the gene editing biotechs that have arisen since CRISPR was pioneered nearly a decade ago. Instead of trying to erase or rewrite portions of a patient’s actual DNA — those As, Ts, Cs and Gs — Chroma will try to change the way that DNA is expressed in the cell.

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Jonathan Weissman, MIT

Ex­clu­sive: Big-name aca­d­e­mics and in­vestors are qui­et­ly prepar­ing a slate of new (epi)genome edit­ing com­pa­nies

A few weeks after Jennifer Doudna introduced CRISPR/Cas9 genome editing to the world, one of her old students decided to take the central part of the biology-altering invention and kill it.

CRISPR/Cas9, as the name implies, is a two-part system: a string of letters called a guide RNA, that says where to cut the DNA. And an enzyme, Cas9, that does the cutting. Often compared to molecular scissors, it was the first system that allowed researchers to cut DNA with ease and precision, promising potential cures for genetic diseases such as sickle cell and cystic fibrosis.

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