Cell/Gene Tx channel feed

3 days ago

BioN­Tech opens new plas­mid DNA man­u­fac­tur­ing fa­cil­i­ty in Ger­many

Ger­man mR­NA play­er BioN­Tech opened the doors to a new man­u­fac­tur­ing fa­cil­i­ty on Thurs­day, this one just about 75 miles north of its head­quar­ters in Mainz, Ger­many.

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FDA places hold on 4D Mol­e­c­u­lar’s Fab­ry gene ther­a­py

2 days ago
FDA+

Man­u­fac­tur­ing roundup: Catal­ent caps off man­u­fac­tur­ing site in Bel­gium; Eu­roAPI restarts pro­duc­tion in Hun­gary

3 days ago
Manufacturing
latest in Cell/Gene Tx

'Moon­shot' at liv­er cell ther­a­py biotech Am­bys Med­i­cines comes to an end

4 days ago
People
Startups

Four years in, Sana gets first FDA go-ahead to bring can­cer treat­ment in­to the clin­ic

Last week
FDA+

J&J, Leg­end say Carvyk­ti beat stan­dard ther­a­py in ear­li­er-line blood can­cer

Last week
R&D
Pharma

Xy­lo­Cor wraps up PhII for heart dis­ease gene ther­a­py, plans for piv­otal tri­al

Last week
R&D

A stem cell pi­o­neer sent an ex­per­i­ment in­to space. Pa­tients are the next fron­tier

Last week
People
In Focus
January 24, 2023 03:14 PM EST
Pharma
Cell/Gene Tx

Can­cer cell-based ther­a­pies com­ing in­to 'im­por­tan­t' year for R&D, say SVB an­a­lysts

Katherine Lewin

News Reporter

Despite investor doubt in allogeneic cell therapy, 2023 will be an “important” year for both autologous and allogeneic immune-cell-based therapies in multiple myeloma and non-Hodgkin’s lymphoma, SVB analysts predict.

With drug candidates like mosunetuzumab, glofitamab and epcoritamab making their way through clinical trials, SVB Securities analysts are predicting the commercialization of bispecific CD3 T-cell engagers will begin to be a part of treatment paradigms as these drugs get approved.

Kenji Yasukawa, Astellas CEO (Akio Kon/Bloomberg via Getty Images)
January 20, 2023 11:09 AM ESTUpdated 01:53 PM
Cell/Gene Tx
FDA+

FDA lifts clin­i­cal hold on Astel­las’ Pompe gene ther­a­py

Lei Lei Wu

News Reporter

The FDA has lifted a clinical hold on Astellas Pharma’s Pompe disease gene therapy trial, the Japanese company announced Friday morning. The FDA put the hold in place last June after investigators flagged a serious case of sensory nerve damage in a patient.

Astellas says it is making strides toward resuming dosing in the Phase I/II clinical trial for adult patients with late-onset Pompe disease, in which they lack an enzyme called GAA that breaks down complex sugars in the body, causing said sugars to build up and damage the muscles. Astellas’ head of regulatory affairs David Smethurst declined to comment on when the trial would resume dosing.

Kleanthis Xanthopoulos, Shoreline Biosciences CEO
January 19, 2023 10:50 AM EST
Startups
Deals
Cell/Gene Tx

Off Ed­i­tas’ shelves, iNK cell ther­a­pies float to Shore­line

Kyle LaHucik

Associate Editor

Editas Medicine is offloading its iNK cell franchise and licensing out some gene editing tech to a private biotech that counts BeiGene and Gilead’s Kite as friends.

Shoreline Biosciences is buying EDIT-202 and another undisclosed iNK program as part of the deal. MiNK Therapeutics had also been vying for the preclinical solid tumor cell therapy, Endpoints News reported last fall.

The San Diego biotech also obtains certain manufacturing tech, an exclusive license to Editas’ so-called SLEEK gene editing knock-in tech and a non-exclusive license to the Boston-area biotech’s engineered AsCas12a enzyme.

Endpoints News

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January 18, 2023 07:00 AM EST
R&D
Cell/Gene Tx

Gene ther­a­py side ef­fects prompt search for new de­liv­ery meth­ods

Max Gelman

Senior Editor

SAN FRANCISCO — Just a few years since the first gene therapies became available, there has been a shift among small biotechs and large pharma companies about how to best deliver the edited genes, in particular regarding the viruses and other vectors that insert gene therapy payloads into cells.

The goal of these new delivery techniques is a more precise mechanism less likely to produce harmful side effects, and the shift is likely to ripple through the sector.

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January 13, 2023 01:28 PM EST
Pharma
Cell/Gene Tx

EMA de­vel­op­ing physi­cian guid­ance for po­ten­tial liv­er dam­age af­ter tak­ing Zol­gens­ma

Katherine Lewin

News Reporter

The European Medicines Agency is working on new advice for healthcare providers treating patients with Zolgensma, five months after two child patient deaths in Russia and Kazakhstan.

That includes monitoring the liver function of patients treated with the gene therapy and quickly assessing cases of suspected liver damage, Reuters first reported.

The EMA released its own statement after the Pharmacovigilance Risk Assessment Committee meeting on Jan. 12:

Devyn Smith, Arbor Biotechnologies CEO
January 11, 2023 10:18 AM EST
Startups
Deals
Cell/Gene Tx

Ver­tex gets more pre­cise with Ar­bor in ex­pand­ed gene edit­ing pact

Kyle LaHucik

Associate Editor

Feng Zhang’s Arbor Biotechnologies is expanding its alliance with Vertex Pharmaceuticals, but is staying mum on financials this time around.

The CRISPR gene editing startup, which has been relatively quiet aside from partnership and capital-raising news since its March 2018 unveiling, is providing its Boston partner with access to precision editing technology for in vivo genetic medicines for “up to three diseases.”

Endpoints News

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David Kirn, 4D Molecular Therapeutics CEO
January 10, 2023 12:49 PM ESTUpdated January 11, 07:57 AM
R&D
Cell/Gene Tx

4D Mol­e­c­u­lar paus­es en­roll­ment in Fab­ry tri­als af­ter kid­ney prob­lems in three pa­tients

Kyle LaHucik

Associate Editor

4D Molecular Therapeutics said it has stopped enrolling patients in its two clinical trials of a gene therapy being investigated for Fabry disease.

The biotech will wait until the second half to evaluate 12-month clinical data on the six patients enrolled on 4D-310. The two trials, one in the US and the other in Taiwan and Australia, were each estimated to enroll 18 adults with the disease, in which the body lacks the enzymes needed to break down fats. For those with the inherited lipid storage disorder, fats build up in blood vessels and tissue, which leads to difficulties in the kidneys, heart and nervous system.

Al Sandrock, Voyager Therapeutics CEO
January 9, 2023 02:01 PM EST
Deals
Cell/Gene Tx

Voy­ager goes big, se­cur­ing mul­ti-bil­lion dol­lar gene ther­a­py de­vel­op­ment deal with Neu­ro­crine

Paul Schloesser

Associate Editor

The neurological disease outfit helmed by Biogen’s former R&D chief announced a big gene therapy deal Monday, starting #JPM23 with its largest deal to date.

Voyager Therapeutics is once again going after gene therapies with Neurocrine Biosciences. The two teamed up on a large pact in 2019, but the Parkinson’s portion of the tie-up fizzled out in February 2021. The work on Friedreich’s ataxia from that deal remains ongoing.

Endpoints News

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JJ Bienaimé, BioMarin CEO
January 9, 2023 11:51 AM ESTUpdated 02:46 PM
R&D
Cell/Gene Tx

Bio­Marin buffs up da­ta on he­mo­phil­ia A gene ther­a­py as FDA de­ci­sion dead­line ap­proach­es

Lei Lei Wu

News Reporter

BioMarin says its Phase III study for a hemophilia A gene therapy met its primary and secondary endpoints at year three, and it has added more data per the FDA’s request. The data come ahead of a March 31 FDA decision deadline.

Last August, the San Rafael, CA biotech’s gene therapy — known as valoctocogene roxaparvovec, or valrox for short — won conditional approval in the EU, where it is marketed as Roctavian and costs about $1.5 million. In the US, BioMarin expects to price the drug between $2 million and $3 million, CEO Jean-Jacques Bienaimé has previously said, which a spokesperson reiterated in an email to Endpoints News.

Endpoints News

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Debanjan Ray, Synthekine CEO
January 6, 2023 08:00 AM ESTUpdated January 9, 04:32 PM
Financing
Startups
Cell/Gene Tx

Syn­thekine nabs $100M to boost IL-2 pro­grams af­ter ri­vals hit set­backs

Kyle LaHucik

Associate Editor

Synthekine reeled in a $100 million financing to bankroll its IL-2 ambitions and get into the clinic with its first CAR-T.

The Series C fundraise will beef up the Merck-partnered biotech’s shot at proving itself in a field that ran into major setbacks last year, when Sanofi took a $1.6 billion hit to its IL-2 work, and Bristol Myers Squibb and collaborator Nektar Therapeutics wound down programs from their $3.6 billion pact.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,600+ biopharma pros reading Endpoints daily — and it's free.

Can­cer cell-based ther­a­pies com­ing in­to 'im­por­tan­t' year for R&D, say SVB an­a­lysts

FDA lifts clin­i­cal hold on Astel­las’ Pompe gene ther­a­py

Off Ed­i­tas’ shelves, iNK cell ther­a­pies float to Shore­line

Gene ther­a­py side ef­fects prompt search for new de­liv­ery meth­ods

EMA de­vel­op­ing physi­cian guid­ance for po­ten­tial liv­er dam­age af­ter tak­ing Zol­gens­ma

Ver­tex gets more pre­cise with Ar­bor in ex­pand­ed gene edit­ing pact

4D Mol­e­c­u­lar paus­es en­roll­ment in Fab­ry tri­als af­ter kid­ney prob­lems in three pa­tients

Voy­ager goes big, se­cur­ing mul­ti-bil­lion dol­lar gene ther­a­py de­vel­op­ment deal with Neu­ro­crine

Bio­Marin buffs up da­ta on he­mo­phil­ia A gene ther­a­py as FDA de­ci­sion dead­line ap­proach­es

Syn­thekine nabs $100M to boost IL-2 pro­grams af­ter ri­vals hit set­backs