Cell/Gene Tx channel feed

William Cao (Bloomberg via Getty)

Nas­daq rings in its first biotech IPOs of 2021: a CAR-T spe­cial­ist from Chi­na, a can­cer port­fo­lio play, and a SPAC

There was once a time when stellar CAR-T data from China would come — in a sense — out of nowhere and be greeted with surprise, or even outright skepticism.

It wasn’t that long ago, but those days are now gone. This is a time a CAR-T therapy originating from China can be developed in the public eye, down to every cut of data, by a company bound by American rules of disclosure.

Case in point: Gracell Biotechnologies, a Shanghai-based outfit that’s grabbing Nasdaq’s first biotech IPO listing alongside Cambridge, MA-based Cullinan Oncology.

FDA of­fers first thoughts on neu­rode­gen­er­a­tive dis­ease gene ther­a­pies

The FDA has issued draft guidance on the development, testing, and trial design for human gene therapies for neurodegenerative diseases. The document, released on Tuesday, also highlights approval pathways for these novel products.

The draft guidance, which applies to products for both adult and pediatric populations, emphasizes the importance of early communication with FDA before the submission of an investigational new drug (IND) application. The agency pointed to INTERACT meetings, which can be used to discuss issues in a product’s early preclinical program, and pre-IND meetings, which occur later in development but prior to the submission of an application.

Martin Silverstein

Mana joins the hec­tic fight against sol­id tu­mors with an ‘off-the-shelf’ can­di­date an­gling for an IND this year

The hunt for effective therapies for solid tumors has heated up in early 2021 with a string of biotechs touting big investor checks and name-brand collaborations to chase those hard-to-treat lumps. Now, with one of its candidates already in the clinic for leukemia, Mana Therapeutics is taking a new round of funding to join the fray.

On Friday, Mana unveiled a $35 million Series A financing round that will help push the Boston-area biotech’s lead candidate through a Phase I trial and could help the company secure an IND for an off-the-shelf allogeneic molecule targeting transplant-ineligible AML and solid tumors within the year.

Rid­ing on rosy es­ti­mates of Zol­gens­ma sales, Re­genxBio sells part of its roy­al­ty for $200M

RegenxBio’s journey as a drug developer may have been less than rosy, but there’s been little doubt about the value of the gene therapy technology it obtained from Jim Wilson’s labs.

Healthcare Royalty Management has made that clear once again, striking a deal to buy a portion of its royalties on Zolgensma for $200 million.

According to the latest quarterly filing from RegenxBio, it’s received $40.7 million in royalties in the first nine months of 2020, compared to just over $10 million in the same period in 2019.

FDA slaps a hold on Voy­ager's lead gene ther­a­py over 'M­RI ab­nor­mal­i­ties'

A rocky year for gene therapy is closing out with more bleak news.

Voyager Therapeutics announced Tuesday that the FDA has forced the company to halt studies on their lead program, a gene therapy for Parkinson’s disease, after “MRI abnormalities” appeared in some patients in a Phase II trial. The hold comes a month after Voyager disclosed that a data safety monitoring board had recommended a pause after “review[ing] certain patient imaging data.”

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Patrick Soon-Shiong, AP

Patrick Soon-Sh­iong merges trou­bled 'moon shot' Nan­tK­west and cell ther­a­py play Im­mu­ni­ty­Bio in late-stage ex­pan­sion

Once one of the hottest brands in biotech, Patrick Soon-Shiong’s next-gen cancer brainchild NantKwest has taken its lumps in recent years with little to show from its natural killer cell platform. Now, with his cell therapy play ImmunityBio moving much faster in the clinic, Soon-Shiong will merge the two companies together to streamline their market aspirations.

NantKwest will reverse merge with Soon-Shiong’s ImmunityBio in a stock-for-stock transaction to bring 11 late-stage natural killer and cell therapy candidates for oncology and infectious diseases under a single roof, the companies said Monday.

Rachel McMinn, Neurogene CEO

Af­ter leav­ing Wall Street to launch a gene ther­a­py up­start, Rachel McMinn nabs $115M to dri­ve her first can­di­date to the clin­ic

When former analyst Rachel McMinn started Neurogene from her apartment around three years ago, she would joke that they’d get office space as soon as her living room table was no longer big enough to hold company meetings.

“We lasted about a year before my living room couldn’t take it anymore,” she said.

With several gene therapies for Batten disease and other lysosomal storage disorders in the preclinical and discovery stage, Neurogene is now bound for the clinic. And on Wednesday, they announced a $115 million Series B to get them there.

Af­ter reel­ing back a gene ther­a­py from Sanofi, a North Car­oli­na up­start bags enough mon­ey to do some­thing about it

After spending some time in Sanofi’s pipeline, a gene therapy for a genetic eye disease that causes blindness is now back in the hands of its creators at Atsena Therapeutics. And on Wednesday, the North Carolina-based startup landed a $55 million Series A to push it through a Phase I/II trial.

Atsena’s founders, power couple Shannon and Sanford Boye, did some early work on the gene therapy at the University of Florida in the early 2000s, before it was licensed by Sanofi. The biotech launched this year with $8 million in funding, then licensed the candidate from Sanofi for an undisclosed amount in July.

Eli Lilly CEO David Ricks

Eli Lil­ly picks up gene ther­a­py play­er Pre­vail in deal worth $1B+, mak­ing good on bolt-on pledge from ear­li­er this year

Back in January, Indiana’s Eli Lilly touted its plan to tack on a suite of mostly early-stage assets in targeted therapeutic areas a la its $1.1 billion pickup of Dermira. In the final days of 2020, Lilly continues to make good on that promise, acquiring a neurodegenerative disease player to flesh out its early-stage pipeline and snag a better foothold in gene therapy.

Lilly will shell out up to $1.04 billion to acquire Prevail, paying $22.50 per share in cash at closing plus a $4 contingent-value right (CVR) based on one of Prevail’s AAV9 gene therapies for neurodegenerative diseases receiving an approval in one of a group of developed nations by Dec. 31, 2024, the companies said in a release Tuesday.

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Jørgen Søberg Petersen, Hemab chairman (Novo Holdings)

No­vo Hold­ings in­vests in Hemab, a com­pa­ny go­ing against the he­mo­phil­ia gene ther­a­py grain with a plan for bis­pecifics

Novo Holdings, the holding company comprising Novo Nordisk and managing the Novo Nordisk Foundation’s assets, has made a new investment through its seed capital arm.

The incubator Novo Seeds is funding a company called Hemab ApS, which is focused on developing bispecific antibodies for rare bleeding disorders. Co-founded by two former Novo Nordisk execs in their hemophilia R&D department, Hemab is aiming for hemophilia to be its first target with related blood diseases to follow afterward.