Cell/Gene Tx channel feed

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

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)

FDA lifts clin­i­cal hold on Astel­las’ Pompe gene ther­a­py

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

Off Ed­i­tas’ shelves, iNK cell ther­a­pies float to Shore­line

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

Keep reading Endpoints with a free subscription

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

Gene ther­a­py side ef­fects prompt search for new de­liv­ery meth­ods

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

EMA de­vel­op­ing physi­cian guid­ance for po­ten­tial liv­er dam­age af­ter tak­ing Zol­gens­ma

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

Ver­tex gets more pre­cise with Ar­bor in ex­pand­ed gene edit­ing pact

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

Keep reading Endpoints with a free subscription

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

David Kirn, 4D Molecular Therapeutics CEO

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

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

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

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

Keep reading Endpoints with a free subscription

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

JJ Bienaimé, BioMarin CEO

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

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

Keep reading Endpoints with a free subscription

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

Debanjan Ray, Synthekine CEO

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

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.