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April: Novartis report on data from the STRONG trial.


23rd June: Novartis announced data confirming benefit of zolgensma in pre symptomatic children (See SPR1NT )

18th January: Study showed Zolgensma can benefit children previously treated with nusinersen – read more.


19th October: Interim data published from the STRONG trial.

21st August: FDA’s partial hold on OAV-101 intrathecal clinical trial STRONG lifted

23rd April: Novartis announced new global trial SMART


23rd September: Following the October 2019 partial hold put on the STRONG trial of the intrathecal (IT) formulation of AVXS-101 in older patients with spinal muscular atrophy, the FDA acknowledged the potential of AVXS-101 IT in this patient population and recommended a pivotal confirmatory study to supplement the existing STRONG data.

19th May: The EC granted approval for Zolgensma® (onasemnogene abeparvovec) for the treatment of babies and young children who have 5q SMA with a bi-allelic mutation in the SMN1 gene and either a clinical diagnosis of SMA Type 1 or up to three copies of the SMN2 gene. Read the European Medicines Agency Report.

27th March: AveXis provided an update on several of their clinical studies (SPR1NT; STR1VE-US and START). The safety data that had accumulated across all trials of Zolgensma from 335 patients indicated that it is generally safe and well tolerated. Nearly all patients experienced adverse events, however, most were not serious and unrelated to the treatment.


30th October: The FDA in the US placed a partial hold on STRONG clinical trials of intrathecal (lumbar puncture) injections of AVXS-101 for SMA patients. This was based on findings in a small pre-clinical animal study.

19th October: Interim data published from the STRONG trial.

7th May: At the 2019 American Academy of Neurology conference in Philadelphia, AveXis presented positive results from clinical trials on Zolgensma, across a broad spectrum of patients with SMA. These were from STRONG, Phase 3 of STR1VE and Phase 3 of SPR1NT.

17th April: Phase 3 clinical trials to date showed prolonged and event-free survival and significant milestone achievement in children who have SMA Type 1. Of the 150+ patients treated with Zolgensma, 5% of screened patients up to 5 years old were excluded due to an immune response against the virus.


27th September: Recruitment had started for the Phase 3 study STR1VE-EU.

25th January: Two pivotal trials of AVXS-101 initiated in the US and EU: STR1VE-US and STR1VE-EU. A fourth clinical study, STRONG was also recently initiated.


15th December: The FDA gave permission for Phase 1 of the STRONG trial.

15th December: Summary and Comparison of Results to date for Nusinersen (ENDEAR Trial) and AVXS-101 Gene Therapy – read more.

12th October: The FDA gave permission for the STR1VE trial.

22nd June: AveXis Gene Therapy Manufacturing Process Streamlined – read more.

5th April: AveXis announced results of Phase 1 START trial.

9th February: AveXis announced European pivotal study STRIVE EU.


7th November: AveXis announced new pivotal study coming – STR1VE US.

18th August: Avexis released data from ongoing START trial.

28th July: The US FDA granted “Breakthrough Therapy” status to AVXS-101, based on early results from the ongoing Phase I, open-label clinical trial. To be granted Breakthrough Therapy status a drug must be designed to treat a serious disease, and show an early indication in clinical trials of being able to provide substantial benefit to patients, both of which apply to AVXS-101.

23rd May: At two separate conferences in the USA in May, Avexis presented the latest data from the ongoing Phase 1 clinical trial of their gene therapy drug for SMA, AVXS-101 – previously known as scAAV9.CB.SMN and ChariSMA (START)

8th January: Avexis announced completion of Phase 1 Trial enrolment (START)


6th January: Study published of SMN Gene Therapy in a large animal model of SMA – read more.


8th October: ChariSMA™, a potential gene therapy for SMA, which has the technical name scAAV9.CB.SMN received Orphan Drug Designation from the FDA .

30th April: Phase I clinical trial to test the safety and efficacy of scAAV9.CB.SMN in Type I SMA children aged 0-9 months announced (START).

15th April: ReGenX Biosciences, LLC and AveXis Inc. agreed an exclusive license for the development of gene therapies to treat SMA using viruses known as NAV® rAAV9 vectors.


23rd October: Gene therapy, scAAV9.CB.SMN, granted Fast Track status by the FDA in the US. This is for drugs that show considerable potential for being able to treat life-threatening diseases that have no available therapeutic options. It means that the development and review processes of a promising drug can be streamlined in order to hopefully speed up translation into the clinic.

12th February: Report from Azzouz Laboratory gene therapy for SMA.


Separate Separate published studies from the laboratories of Dr. Brian Kaspar, Dr. Martine Barkats, and Prof. Mimoun Azzouz show that the adeno-associated virus type 9 (AAV9) is able to cross the blood-brain barrier and produce SMN protein, resulting in drastic improvements in SMA model mice – read more.