19:47 , Aug 14, 2019 |  BC Extra  |  Company News

Top R&D execs out at AveXis

AveXis said Wednesday that top R&D executives were pushed out of operations in early May and have left the company, the first revelations of employee repercussions after the pharma revealed it knew that data for...
23:48 , Aug 13, 2019 |  BC Extra  |  Company News

Aug. 13 Company Quick Takes: Pharming licenses Novartis' CDZ173; plus Novartis, Merck-Harvard, Mustang, J&J

Pharming licenses Novartis autoimmune candidate  Pharming Group N.V. (Euronext:PHARM) gained rights to PI3Kδ inhibitor CDZ173 from Novartis AG (NYSE:NVS; SIX:NOVN) for patients with activated PI3Kδ syndrome, a primary immune deficiency. Novartis will receive $20 million...
08:06 , Aug 13, 2019 |  BC Extra  |  Company News

Via Synpromics takeout, AskBio adds promoters to gene therapy platform

With the acquisition of Synpromics, well-funded gene therapy developer AskBio has added synthetic promoter technology to its toolkit as it prepares to begin clinical testing of four candidates by YE20. The takeout comes about three...
21:59 , Aug 12, 2019 |  BC Extra  |  Politics & Policy

Aug. 12 P&P Quick Takes: China to implement ethics committee; plus Senators call for action on AveXis and FDA’s Fabry guidance

China planning national science, technology ethics committee  China’s central committee for deepening overall reform approved a plan to establish a national science and technology ethics committee. The government said the committee is meant to improve...
03:21 , Aug 10, 2019 |  BioCentury  |  Regulation

Timeline of a debacle: Novartis missteps hold lessons for diligence, new modalities

Aside from all the questions about who knew what when, and how they should have acted, the Zolgensma data fiasco reveals that Novartis’ due diligence team missed some warning signs. The case has the hallmarks...
02:14 , Aug 10, 2019 |  BioCentury  |  Product Development

Payers’ view of reimbursement when gene therapy isn’t enough

Spinal muscular atrophy is giving payers a new conundrum, and the indication is poised to become a testing ground with implications for many if not all gene therapies. The issue is whether, and how, payers...
21:23 , Aug 7, 2019 |  BC Extra  |  Company News

Novartis resolute that it acted in good faith on Zolgensma

Novartis contends that the three-month lag between when it discovered Zolgensma data were manipulated and when it reported the issue to FDA was justified and didn't put patients at risk. However, the pharma is doing...
19:00 , Aug 6, 2019 |  BC Extra  |  Company News

FDA: AveXis manipulated potency data for Zolgensma

FDA said Tuesday that AveXis manipulated in vivo potency data for gene therapy Zolgensma and failed to report the issue to the agency, holding back the information while the agency was reviewing its BLA. The...
00:57 , Jul 19, 2019 |  BC Extra  |  Company News

Payers, patients coming on board for Novartis’ Zolgensma

Novartis reported steady uptake for newly launched gene therapy Zolgensma, with about 40% of lives reimbursed via formal coverage policies and 17 payers signing up for the pharma’s novel pricing arrangement. But payers are resisting...
00:22 , Jul 2, 2019 |  BC Extra  |  Clinical News

Biogen's Spinraza yields long-term preservation of motor development in SMA

Gearing up to compete with a gene therapy in SMA, Biogen presented long-term data from its antisense therapy Spinraza that could help make the case for continued use of the chronic treatment. In the open-label...