SEC Filings

AVEXIS, INC. filed this Form 10-K on 02/28/2018
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cGMP‑compliant manufacturing facility. Based on data from our intended current cGMP development work and feedback from ongoing discussions with the FDA, including our chemistry, manufacturing and controls, or CMC, meeting with the FDA in May 2017, we made the strategic decision to use our intended cGMP derived product in all current and future studies of AVXS‑101 and for all commercial needs if AVXS-101 receives marketing approval. We are executing on our manufacturing plan to ensure there is sufficient supply to meet our needs for both clinical material and for commercial demand, if AVXS-101 is approved by the FDA. We will continue to evaluate our internal manufacturing capabilities and engage third-party manufacturers, if needed, to support production and provide alternate supply for both AVXS-101 and for our other programs.


Invest in developing and accessing intellectual property to further expand our product portfolio.  To date, we have secured our intellectual property position through our agreements with our key collaborators and other third parties. We plan to build upon this intellectual property position through additional patent applications related to AVXS‑101. With respect to future product candidates, we expect to continue to work with REGENXBIO and other creators of next generation vectors to ensure appropriate access to additional therapeutic candidates.


Continue to develop a strong, collaborative network of key stakeholders, including patient advocacy groups, healthcare professionals, key opinion leaders and research institutions, to inform our clinical development and commercialization strategies.  We believe that it is imperative to put the patient at the center of our focus, and we intend to continue to work and listen closely to key stakeholders to ensure that we clearly understand their issues, insights and recommendations. The feedback from and collaboration with these groups have and will continue to inform our key strategies to transform the lives of patients and their families suffering from rare and life‑threatening neurological genetic diseases with safe and effective therapies.


Build organizational capabilities to establish commercial readiness to market AVXS-101 once regulatory approval is received. We are in the process of building a commercial infrastructure capable of launching AVXS-101 in the United States. This involves understanding the market, determining strategies to make the therapy accessible to physicians and patients once approved, and hiring the necessary personnel.

Background on Gene Therapy

Many diseases are driven by genetic mutations in which the mutated genes can affect the production of proteins. Gene therapy attempts to address disease biology by introducing recombinant DNA into a patient’s own cells, commonly in the form of a functional copy of the patient’s defective gene, to address the genetic defect and modulate protein production and cellular function, which provides therapeutic benefit.

Using gene therapy, physicians can introduce or re‑introduce genes that encode for a therapeutic protein. Instead of providing proteins or other therapies externally and dosing them over a long period, we believe gene therapy offers the possibility of dosing a patient once to achieve a long‑term, durable benefit. Gene therapies are typically comprised of three elements: a transgene, a promoter and a delivery mechanism such as our AAV9 capsid. Once the therapeutic gene is transferred to a patient’s cells, we believe the cells may be able to continue to produce the therapeutic protein for years or, potentially, the rest of the patient’s life. As a result, gene therapy has the potential to transform the way these patients are treated by addressing the underlying genetic defect.

Background on Spinal Muscular Atrophy

SMA is a severe neuromuscular disease characterized by the loss of motor neurons leading to progressive muscle weakness and paralysis. The incidence of SMA is approximately one in 10,000 live births, and one in 50 people are carriers of the SMA gene (approximately six million Americans). SMA is generally divided into sub‑categories termed SMA Type 1, 2, 3 and 4. SMA, and the SMA sub‑types, the genetic diagnosis is made by first identifying the existence of a genetic defect in the SMN1 gene and then determining the number of copies of the SMN2 backup gene, which correlates with disease onset and severity. If insufficient protein is expressed, muscles do not develop properly.



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