Pharming Group drug Joenja was awarded FDA approval for treating a primary immunodeficiency called activated phosphoinositide 3-kinase delta syndrome, or APDS. It’s the first treatment for the rare disorder.
RNA therapies developer ProQR Therapeutics is eying a future without ophthalmology. After receiving European Medicines Agency feedback for its lead program for a rare eye disorder, the biotech has decided to find a strategic partner for all of its eye drug assets and apply its RNA technology to other therapeutic areas.
Munck Wilson Mandala Partner Greg Howison shared his perspective on some of the legal ramifications around AI, IP, connected devices and the data they generate, in response to emailed questions.
Targeting mutations isn’t the only way to treat genetically driven diseases. Scenic Biotech is developing molecules to target genes that suppress disease, and it has raised $31 million to advance its pipeline of potential therapies for cancer and rare diseases.
The lead RNA therapy from ProQR Therapeutics has failed the main and secondary goals of a pivotal study testing it as a treatment for a rare, inherited vision disorder. Despite the disappointing preliminary data, the biotech said it remains confident in the technology that produced the experimental therapy, sepofarsen, and the Phase 2/3 study will continue.
Myasthenia gravis patients now have a new FDA-approved biological treatment. Argenx drug Vyvgart provides a treatment alternative for patients who have the rare autoimmune disorder and also marks the first approved product in a new class of medicines.
A uniQure gene therapy for hemophilia B in on track for an FDA submission in the first quarter of 2022. Meanwhile, uniQure added to its pipeline with the acquisition of preclinical epilepsy gene therapy biotech Corlieve Therapeutics for €46.3 million upfront.
At the Abarca Forward conference earlier this year, George Van Antwerp, managing director at Deloitte, discussed how social determinants of health and a personalized member experience can improve medication adherence and health outcomes.
Cancer drug developer Merus has encouraging early results indicating its drug can address several different types of tumors characterized by NRG1, a rare gene fusion. The company believes the data could support a regulatory filing for the bispecific antibody drug.
ProQR Therapeutics has encouraging data from an early-stage study evaluating its treatment for vision loss in the rare, inherited disorder Usher syndrome. The biotech now plans to advance its RNA therapy to two pivotal studies later this year.
The company plans to start clinical development next year. Its focus is on bispecific antibodies - a form of cancer immunotherapy - that use gamma-delta "surveillance" T cells.
The therapy, etranacogene dezaparvovec, is currently in Phase III clinical development for hemophilia B. Shares of uniQure were down nearly 20%, but an investment bank analyst wrote that the financial terms were favorable for the company.
We will highlight Build My Health's revenue practice management tools, which could help physician practices add up to $250,000 to their practices.
The company presented updated data for the drug, voxelotor, at the European Hematology Association conference in Amsterdam Friday.
On Saturday, the company will present a poster of Phase I trial data for belantamab mafodotin, which targets the same antigen, BCMA, as Amgen bispecific antibody AMG 420 and multiple CAR-T cell therapies in development.
The Financial Times reported Monday that the Japanese drugmaker would merge its UK operations with a Dutch subsidiary.
Many supporters of the UK's withdrawal from the EU say "no deal, no problem," but drugmakers' actions suggest "no deal, big problem."
The Fitch Solutions report, released this week, notes that the number of clinical trials taking place in the UK has already fallen more than 25 percent.