As the FDA closed out day 1 of its marathon gene therapy hearing, the agency gave an unusual glimpse into their conversations — and conflict — with companies trying to start gene therapy trials.

Routinely, FDA official Andrew Byrnes explained in the only followup question regulators asked during the 8-hour session, the agency has been forced to grapple with an impurity known as empty capsids — a long known but little discussed potential safety issue for gene therapies — and, crucially, how much to push back on companies who refuse to remove them because of how costly or time consuming it might be.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,600+ biopharma pros reading Endpoints daily — and it's free.

SIGN UP LOG IN

Audentes’ troubled the gene therapy program for a rare and devastating muscle wasting disease isn’t yet out of the woods.

The Astellas subsidiary has once again halted a trial for its X-linked myotubular myopathy candidate, the Japanese Pharma announced, after a serious adverse event emerged in which researchers observed “abnormal liver function tests” within the first month of dosing. The hold is voluntary for now, but Astellas left open the possibility of receiving an official FDA notice.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,600+ biopharma pros reading Endpoints daily — and it's free.

SIGN UP LOG IN

Ten months after the FDA took the shackles off Poseida’s study for a CAR-T directed against castrate-resistant prostate cancer, the executive team is now rolling out an early snapshot of the proof-of-concept data it’s looking for in a small, early-stage study.

Tested in 9 heavily pre-treated patients — with an average of 6 prior therapies — investigators tracked a significant, 50%-plus drop in PSA levels in 3 of those patients and what they called a “concordant” imaging result for tumor effect. One patient had a complete response — the ideal result for any cancer study.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,600+ biopharma pros reading Endpoints daily — and it's free.

SIGN UP LOG IN

As one of the pioneers behind CRISPR, the Broad Institute’s Feng Zhang has fashioned himself into a gene editing Renaissance man in recent years with a finger in every pie. For RNA editing, one of his passion projects, Zhang has worked to crack a chronic delivery logjam — and his team has just made a big breakthrough there.

A team of researchers out of Zhang’s lab has built a pair of “ultracompact” RNA editing tools that can fit inside an adeno-associated virus (AAV) package, potentially creating a breakthrough in the delivery of those gene editing tools into human cells, according to an article published in Nature Biotechnology on Monday.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,600+ biopharma pros reading Endpoints daily — and it's free.

SIGN UP LOG IN

With the success of Moderna, parent company Flagship Pioneering has become the mothership for even more audacious next-gen platform plays. Investors like what they see, and now they’ve opened the vault to a particularly bold Flagship startup working on ring-shaped RNA it thinks could be the future of drugmaking.

Laronde has closed a $440 million Series B — one of the largest ever fundraising rounds of its type — to pursue a programmable RNA platform the biotech has previously said could churn out 100 marketed drugs or drug programs in 10 years.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,600+ biopharma pros reading Endpoints daily — and it's free.

SIGN UP LOG IN

A new twist emerged Thursday in the long-running patent fight between Juno Therapeutics and Gilead’s Kite Pharma over CAR-T technology, one that gets Kite off the hook for more than $1.2 billion in damages.

The US Court of Appeals for the Federal Circuit reversed a previous decision awarding the money to Juno, ruling in a 19-page opinion that a patent owned by the Bristol Myers Squibb subsidiary and Sloan Kettering is invalid. All three panel judges agreed with Kite’s position, overturning a verdict from December 2019 after finding “substantial evidence does not support the jury’s verdict in Juno’s favor.”

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,600+ biopharma pros reading Endpoints daily — and it's free.

SIGN UP LOG IN

Novartis’ efforts to move its CAR-T into earlier lines of treatment have hit a brick wall.

Kymriah, its pioneering CD19-targeting therapy, has failed to bend the curve on event-free survival for patients with second-line aggressive B-cell non-Hodgkin lymphoma when compared with standard of care. While the treatment arm was given infusions of engineered T cells, the SOC group had salvage chemotherapy followed — in responding patients — by high-dose chemotherapy and stem cell transplant.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,600+ biopharma pros reading Endpoints daily — and it's free.

SIGN UP LOG IN