The genome editing revolution will be patented. Or at least that’s the case for this latest US patent, awarded Tuesday to the Broad Institute’s David Liu and team for the compositions and methods behind the prime editing of a target DNA molecule (e.g., a genome) that enables a nucleotide change and/or targeted gene mutation, according to the abstract.

Developed in Liu’s lab at the Broad Institute, which famously defeated Jennifer Doudna and UC Berkeley in court over CRISPR-related patents, the idea behind prime editing — dubbed CRISPR 3.0 — was published in Nature in December 2019, and that spawned a startup known as Prime Medicine.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,300+ biopharma pros reading Endpoints daily — and it's free.

SIGN UP LOG IN

Renovacor wanted to be in the clinic already. The biotech expected an $11 million Series A in summer 2019 would bankroll an IND filing for its cardiac gene therapy, but as it goes with drug R&D, the path took some twists.

Fifty-four weeks after shipping up to New York City for a blank check merger, Renovacor is turning off the lights on $RCOR and going the way of $RCKT, or Rocket Pharmaceuticals, in an all-stock deal that adds to the latter’s gene therapy ambitions, which includes two likely BLA filings next year.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,300+ biopharma pros reading Endpoints daily — and it's free.

SIGN UP LOG IN

Relative to the protracted journey of developing a gene therapy delivered by a lentiviral vector, the speed at which bluebird bio moved from having one approved product to two was extremely fast.

Exactly a month after the FDA gave its green light to bluebird’s Zynteglo — to treat beta thalassemia — the agency came through with an approval for a second gene therapy for early, active cerebral adrenoleukodystrophy (CALD). The drug, previously known as eli-cel and now branded Skysona, will have a wholesale acquisition price of $3 million and should become available by the end of this year.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,300+ biopharma pros reading Endpoints daily — and it's free.

SIGN UP LOG IN

On the hunt to improve upon the foundations of Luxturna by creating a gene therapy that it hopes can be given to a broader swath of patients with a rare retinal disease than its Big Pharma rival, SparingVision has secured another round of financing. But it didn’t come easy.

The Paris and Philadelphia biotech’s leadership began the Series B process in January, and at that time, they agreed that if by the end of spring it proved to be “too challenging, impossible or that the value that we were looking for was just impossible to obtain, we were going to focus on an insider-only round,” CEO and president Stéphane Boissel told Endpoints News.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,300+ biopharma pros reading Endpoints daily — and it's free.

SIGN UP LOG IN

Four years ago, Cambridge-based Rubius Therapeutics was having the time of its life. The company, which makes engineered red cells, was raking in ample cash and had enjoyed a $1.8 billion market valuation registered at the IPO.

Today it is a whole different story.

Rubius has decided to scrap two of its leading ongoing trials, let go of 75% of its workforce and explore the sale of its manufacturing facility in Rhode Island, the biotech announced Tuesday.

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,300+ biopharma pros reading Endpoints daily — and it's free.

SIGN UP LOG IN