Swiss drugmaker Roche recorded impairment charges of 2.8 billion Swiss francs, or roughly $3 billion, in 2022 to account for falling sales forecasts for marketed cancer drugs as well as for gene therapies acquired via its 2019 deal for Spark Therapeutics.
DORVAL, QC, Sept. 20, 2022 /CNW/ - Novartis Pharmaceuticals Canada Inc. and the pan Canadian Pharmaceutical Alliance (pCPA) have successfully concluded negotiations for Luxturna® (voretigene neparvovec), a one-time gene therapy for the treatment of adult and pediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations.
Co-founder and Chief Scientific Officer Derek Jantz, Ph.D., kicked off the event with a deep dive into the in vivo therapies Precision is developing to address four different diseases, and then he took questions from Wall Street analysts. Much of Jantz’s presentation, and several of the questions that followed, focused on whether the company’s technology could adequately ameliorate the risk of off-target gene editing—inadvertent changes to patients’ DNA that could cause dangerous side effects.
She planted a seed that led to vision loss gene therapy Luxturna, and now Jean Bennett, M.D., Ph.D., is behind another retinal gene therapy biotech. Opus Genetics emerged Wednesday with its own seed in the form of $19 million in funding.
Horama refuels with a Series B expansion and a new name
The NHS has reported treating its first patient with Novartis’ Luxturna (voretigene neparvovec) a “revolutionary” new gene therapy that can restore eyesight, as part of its NHS Long Term Plan.
Inever thought I’d see the day when words like “process,” “scale,” and “automation” would make news in the biopharma industry. Yet as the race heats up to bring more first-of-their-kind gene therapies to market, breakthroughs in manufacturing are often the key — or break down the barrier — to delivering these therapies to patients.
The U.S. Food and Drug Administration is investigating whether the popular heartburn drug Zantac causes carcinogens to form in the bodies of users, in an effort to fully understand the risks posed by the already recalled drug, the agency’s spokesman said on Thursday. The issue of whether ranitidine, commonly known as Zantac, causes levels of the probable carcinogen N-nitrosodimethylamine (NDMA) to rise in users’ bodies has been raised previously by Valisure, an online pharmacy that originally flagged the potential contamination of ranitidine to the FDA.
Patients in a Phase 1/2 trial taking ProQR's experimental eye drug sepofarsen had eyesight improvements gained at three months sustained through one year, giving the company confidence that a Phase 3 trial measuring improvements at 12 months will show similar benefits.
In what should come as no surprise, Roche and Spark Therapeutics have further delayed their plan to merge, with antitrust concerns still being blamed. This week, Roche said it extended its tender-offer period for outstanding shares of Spark to Oct. 1 from Sept. 3.