Cell/Gene Tx channel feed

Samit Hirawat. Bristol-Myers Squibb

December 9, 2019 04:35 AM ESTUpdated 05:36 PM

John Carroll

Bristol-Myers is making a bee-line to the FDA with positive liso-cel data — but is it too late in the CAR-T game?

Bristol-Myers Squibb came to ASH this past weekend with a variety of messages on the new cancer drugs they had acquired in the big Celgene buyout, including liso-cel, the lead CAR-T program picked up in the $9 billion Juno acquisition. And one of the most important was that they had the pivotal efficacy and safety data needed to snag an approval from the FDA next year, with the BLA on track for a filing this month.

Sangamo CEO Sandy Macrae

December 8, 2019 10:11 AM ESTUpdated December 9, 04:21 PM

John Carroll

Cell/Gene Tx

R&D

Patient #9 has been a concern, but Sangamo and Pfizer are bullish about winning the marathon hemophilia A gene therapy race

Patient number 9 has given Sangamo and its partners at Pfizer some heart palpitations in their high profile hemophilia A gene therapy program.

After watching his Factor VIII level rise following treatment like the rest, the crucial efficacy gauge they track saw a sudden and significant plunge. At week 13, the FVIII level had dropped below normal. Then it began to rise again.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,000+ biopharma pros reading Endpoints daily — and it's free.

December 7, 2019 12:34 PM ESTUpdated December 9, 04:17 PM

John Carroll

Cell/Gene Tx

R&D

J&J team shows off 'breakthrough' BCMA CAR-T data, and that could cause a big headache at bluebird and Bristol-Myers

Just hours after J&J’s oncology team bragged about scoring a breakthrough therapy designation for their BCMA CAR-T drug, they pulled the wraps off of the multiple myeloma data for JNJ-4528 that impressed the FDA. And it’s easy to see why they may well be on a short path to a landmark approval — which may well be making the rival team at bluebird/Bristol-Myers more than a little nervous.

Following up on some attention-grabbing data initially presented by their Chinese development partners at Legend 2 years ago during ASCO, investigators say they tracked some jaw-dropping results that confirmed the early promise of a therapy that’s shaking up a crowded field led by bluebird bio.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,000+ biopharma pros reading Endpoints daily — and it's free.

December 6, 2019 08:02 AM EST

Amber Tong

Cell/Gene Tx

R&D

With FDA review of its gene therapy underway, Ferring's $570M spinout delivers in PhIII bladder cancer readout

FerGene was born ready. By the time Ferring spun out the biotech with a gene therapy licensed from FKD Therapies Oy and $400 million in cash from Blackstone, they had secured a priority review on their BLA and pointed to an imminent date for the pivotal Phase III readout in bladder cancer.

The date came yesterday, and FerGene said its experimental drug did not disappoint.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,000+ biopharma pros reading Endpoints daily — and it's free.

ENDPOINTS CAREERS

Scientist- Immunology

Recursion Pharmaceuticals

Salt Lake City, UT

view job offer post your job now

November 27, 2019 10:44 AM EST

Jason Mast

Cell/Gene Tx

Novartis, Vertex outline big gene therapy plans — fueling a global boom

At an old Amgen facility tucked just beyond the Rockies. In a warehouse behind a Walmart supercenter in Durham, North Carolina. On a long-time Bristol Myers Squibb site outside Princeton. The tech has emerged, and now the arms race to physically build a generation of gene therapies has begun.

Novartis will spend $500 million scaling its gene therapy manufacturing efforts, Reuters reported today. That’ll put it nearly on par with Pfizer, who committed $600 million for its facilities even before any of its gene therapies have been approved. Together, 11 companies Reuters surveyed will spend $2 billion on gene therapy production.

Additionally, the Boston Globe reported today that Vertex had completed its search for a gene therapy research and manufacturing campus in Boston, settling on a 256,000 square-foot center at the Raymond Flynn Marine Industrial Park.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,000+ biopharma pros reading Endpoints daily — and it's free.

November 19, 2019 07:06 AM ESTUpdated 10:59 AM

Natalie Grover

Cell/Gene Tx

R&D

In a landmark first glimpse of human data from Vertex, CRISPR/Cas9 gene therapy signals early benefit

Preliminary data on two patients with blood disorders that have been administered with Vertex and partner CRISPR Therapeutics’ gene-editing therapy suggest the technology is safe and effective, marking the first instance of the benefit of the use of CRISPR/Cas9 technology in humans suffering from disease.

Patients in these phase I/II studies give up peripheral blood from which hematopoietic stem and progenitor cells are isolated. The cells are tinkered with using CRISPR/Cas9 technology, and the edited cells — CTX001 — are infused back into the patient via a stem cell transplant. The objective of CTX001 is to fix the errant hemoglobin gene in patents with two blood disorders: beta-thalassemia and sickle cell disease, by unleashing the production of fetal hemoglobin.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,000+ biopharma pros reading Endpoints daily — and it's free.

November 14, 2019 11:27 AM ESTUpdated 11:42 AM

Amber Tong

Cell/Gene Tx

Deals

Sarepta picks up a slate of preclinical gene therapy programs, seeding new StrideBio deal with $48M upfront

Having tasted early success in treating a number of muscular dystrophies with gene therapy, Sarepta is setting itself up to tackle more rare diseases.

The Boston-based biotech has picked StrideBio for a partnership evidently designed to run years, kicking things off with $48 million upfront and four central nervous system targets. The smaller partner is tasked with all R&D work up to the IND stage, coming up with the AAV-based therapies to be tested in Rett syndrome (MECP2), Dravet syndrome (SCN1A), Angelman syndrome (UBE3A) and Niemann-Pick (NPC1).

November 12, 2019 07:15 AM ESTUpdated 07:35 AM

John Carroll

Cell/Gene Tx

R&D

FDA puts Solid Bio’s lead gene therapy program on hold — again — after another patient is hurt by SGT-001

Solid Biosciences continues to be plagued by safety issues.

Close to 18 months after the gene therapy biotech was able to quickly shed an FDA hold on their lead Duchenne muscular dystrophy program for SGT-001, regulators have stepped back in to force another halt after another patient was hit hard by a set of serious adverse events remarkably similar to the first set.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,000+ biopharma pros reading Endpoints daily — and it's free.

ENDPOINTS CAREERS

Senior Biologist, Drug Discovery

PsychoGenics

Paramus, NJ

view job offer post your job now

November 11, 2019 10:22 AM ESTUpdated 10:54 AM

Amber Tong

Cell/Gene Tx

Regulatory

Decrying 'arbitrary and capricious' action, RegenxBio sues FDA over clinical holds on gene therapy

When RegenxBio disclosed that the FDA had placed a partial clinical hold on one of its lead gene therapies, execs outlined several customary next steps: continuing assessment and monitoring, delaying a related IND filing, and working with the FDA to address the matter.

As it turned out, they were planning something much less mundane. Two days after announcing the hold in its Q3 update, RegenxBio filed a lawsuit seeking to set it aside, the FDA Law Blog noted.

November 7, 2019 07:11 AM EST

Amber Tong

Cell/Gene Tx

Regulatory

RegenxBio runs into partial hold for anti-VEGF gene therapy as FDA investigates delivery device

Issues with a delivery device are slowing down RegenxBio’s clinical plans for one of its lead gene therapies.

In its Q3 update, RegenxBio informed investors of a partial clinical hold the FDA has slapped on RGX-314 while regulators review certain third-party commercially-available devices the biotech uses to deliver its treatment. As a result, RegenxBio won’t be able to start its planned Phase IIb trial for wet age-related macular degeneration or file an IND for a Phase II diabetic retinopathy study until early 2020.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 70,000+ biopharma pros reading Endpoints daily — and it's free.

Bris­tol-My­ers is mak­ing a bee-line to the FDA with pos­i­tive liso-cel da­ta — but is it too late in the CAR-T game?

Pa­tient #9 has been a con­cern, but Sang­amo and Pfiz­er are bull­ish about win­ning the marathon he­mo­phil­ia A gene ther­a­py race

Keep reading Endpoints with a free subscription

J&J team shows off 'break­through' BC­MA CAR-T da­ta, and that could cause a big headache at blue­bird and Bris­tol-My­ers

Keep reading Endpoints with a free subscription

With FDA re­view of its gene ther­a­py un­der­way, Fer­ring's $570M spin­out de­liv­ers in PhI­II blad­der can­cer read­out

Keep reading Endpoints with a free subscription

Recursion Pharmaceuticals

Salt Lake City, UT

No­var­tis, Ver­tex out­line big gene ther­a­py plans — fu­el­ing a glob­al boom

Keep reading Endpoints with a free subscription

In a land­mark first glimpse of hu­man da­ta from Ver­tex, CRISPR/Cas9 gene ther­a­py sig­nals ear­ly ben­e­fit

Keep reading Endpoints with a free subscription

Sarep­ta picks up a slate of pre­clin­i­cal gene ther­a­py pro­grams, seed­ing new Stride­Bio deal with $48M up­front

FDA puts Sol­id Bio’s lead gene ther­a­py pro­gram on hold — again — af­ter an­oth­er pa­tient is hurt by SGT-001

Keep reading Endpoints with a free subscription

PsychoGenics

Paramus, NJ

De­cry­ing 'ar­bi­trary and capri­cious' ac­tion, Re­genxBio sues FDA over clin­i­cal holds on gene ther­a­py

Re­genxBio runs in­to par­tial hold for an­ti-VEGF gene ther­a­py as FDA in­ves­ti­gates de­liv­ery de­vice

Keep reading Endpoints with a free subscription

Bristol-Myers is making a bee-line to the FDA with positive liso-cel data — but is it too late in the CAR-T game?

Patient #9 has been a concern, but Sangamo and Pfizer are bullish about winning the marathon hemophilia A gene therapy race

J&J team shows off 'breakthrough' BCMA CAR-T data, and that could cause a big headache at bluebird and Bristol-Myers

With FDA review of its gene therapy underway, Ferring's $570M spinout delivers in PhIII bladder cancer readout

Novartis, Vertex outline big gene therapy plans — fueling a global boom

In a landmark first glimpse of human data from Vertex, CRISPR/Cas9 gene therapy signals early benefit

Sarepta picks up a slate of preclinical gene therapy programs, seeding new StrideBio deal with $48M upfront

FDA puts Solid Bio’s lead gene therapy program on hold — again — after another patient is hurt by SGT-001

Decrying 'arbitrary and capricious' action, RegenxBio sues FDA over clinical holds on gene therapy

RegenxBio runs into partial hold for anti-VEGF gene therapy as FDA investigates delivery device