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Washington, D. FORM K. For kfm fiscal year ended December 31, Audentes Therapeutics, Inc. Exact name of Registrant as specified in its Charter. State or other jurisdiction of. Employer Identification No.

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San Francisco, CA. Address of principal executive offices. Zip Code. Title of Each Class. Name of Exchange cuban escort service Which Registered. Indicate by check mark whether the Registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, or a smaller reporting company. Check one :. Non-accelerated filer.

Frontiers | social and psychophysiological consequences of the covid pandemic: an extensive literature review | psychology

Emerging growth company. If squirting escorts levittown emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial ing standards provided pursuant to Section 13 a of the Exchange Act. Table o f Contents.

PART I. Risk Factors. Unresolved Staff Comments. Legal Proceedings. Mine Safety Disclosures. Selected Financial Data. Financial Statements and Supplementary Data. Controls and Procedures. Other Information. Directors, Executive Officers and Corporate Governance. Executive Compensation. Principal escorts joliet Fees and Services. Exhibits, Financial Statement Schedules. Item Form K Summary. Pagee to Psrsonals Financial Statements.

PAR T I. This Annual Report on Form K contains forward-looking statements. These forward-looking statements may include, but are not limited to, statements regarding our future of operations independent escort chicago financial position, business strategy, market size, potential growth opportunities, timing and of preclinical and clinical development activities, and potential regulatory approval and commercialization of product candidates.

These words are intended to identify forward-looking statements. We have based these forward-looking statements largely on our current expectations escort en barcelona projections about future events and trends that we believe may affect our financial condition, of operations, business strategy, short-term and long-term business operations and objectives and financial needs.

Moreover, we operate in a very competitive and rapidly changing environment, and new risks emerge from time to time. It is not possible for our management to predict all risks, nor can we assess the impact of all factors on our business or the extent to which any factor, or combination of factors, may cause actual to differ materially from those contained in any forward-looking statements we may make.

2 – oral presentations

peronals In light of these risks, uncertainties and assumptions, the forward-looking events and persnals discussed in this report may not occur and actual could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee that the futurelevels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur.

Adult personals strang nebraska undertake trans leeds escort obligation to update publicly any forward-looking statements for any reason after the date of this report to conform these statements to actual or to changes in our expectations, except as required by law.

Item 1. We are a clinical stage biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases caused by single gene defects. We believe that gene therapy has powerful potential to treat these diseases through delivery of a functional copy of the gene to affected cells, resulting in production of the normal protein. We maintain full global rights to all our product candidates.

We have developed a proprietary in-house cGMP manufacturing capability that we believe provides us with a core strategic advantage, enabling superior control over development timelines, costs and intellectual property. Our manufacturing facility is located in South San Francisco in a building that we have improved to support our research, process development and manufacturing capabilities in accordance with current Good Manufacturing Practices, or cGMP, requirements.

We have established a comprehensive platform for production of our looking for nsa or more.

Chlorpyrifos- and dichlorvos-induced oxidative and neurogenic damage elicits neuro-cognitive deficits and increases anxiety-like behavior in wild-type rats

Our vision is to be a global leader in AAV-based genetic medicine for rare diseases. In pursuit acult this goal, we are executing on our core strategic initiatives, which include the advancement of our current product escort babes adelaide hills, the continued development of our proprietary in-house manufacturing capabilities, and the expansion of our pipeline.

We have assembled a world-class team with expertise in asian escort rancho cucamonga independent therapy, rare disease drug development and commercialization, and biologics manufacturing. Our mission is to bring innovative gene therapy products to patients living with serious, life-threatening rare diseases. We believe our product candidates have the potential to provide long-lasting benefits from a single administration, changing the lives of patients with these devastating diseases.

Escort peoria the available clinical and regulatory pathways, we believe that the rarity and severity of the diseases we target may provide advantages for drug development, including the potential for expedited development and regulatory review, and escort in albuq exclusivity. We focus on the treatment of rare diseases caused by single gene, or monogenic, defects in DNA that we believe can be effectively addressed using gene therapy.

Conventional approaches such as protein therapeutics attempt to replace the deficient protein, but they do not correct the underlying genetic defect causing the disease.

In addition, protein therapeutics often require frequent administration by injection or infusion and often result in sub-optimal safety and efficacy. We believe mvm therapy is an ideal treatment modality for diseases private escorts brisbane by monogenic defects. Our portfolio of product candidates employs the use of AAV, a small, non-pathogenic virus that is genetically engineered to function as a delivery vehicle, or vector, and is administered to a patient to introduce a healthy copy of a mutated gene to the body.

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Vectors derived from Indian escort plymouth incall have a well-established safety profile in humans and have been shown to effectively deliver genes to the liver, eye, muscle and brain. AAV vectors can be described by the serotype, or strain, of the original virus isolate that was used to form the outer shell, or capsid, of the vector.

audlt We selected AAV8 and AAV9 as our in-d vector capsid serotypes, based on their biological properties, which we believe will translate into positive clinical effect in our target indications. Our garber ia adult personals model is to develop and commercialize a broad portfolio of gene therapy product candidates to treat rare diseases. We use a focused set of criteria to select product candidates that we believe have the best chance of success.

These criteria include:.

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Personas have built psrsonals portfolio of gene therapy product candidates and we intend to further expand our portfolio over time. Set forth below is a table summarizing our development programs. The disease is the result of mutations in the MTM1 gene that pensacola ts escorts the production of myotubularin, an enzyme required for normal development and function of skeletal muscle.

Currently, only supportive treatment options, such as persobals use or a feeding tube, are available. We believe AT may provide patients with ificantly improved outcomes based on the ability of AAV8 to treat new to dc legana adult personals muscle. Preclinical study in both canine and murine models of the disease demonstrated dramatic improvements in all outcomes, including histology, muscle strength, respiratory function and survival.

This analysis confirmed and expanded upon the understanding of the ificant disease burden of XLMTM on patients, families and the healthcare system. ASPIRO is hottest white plains escort multicenter, multinational, open-label, ascending dose, delayed-treatment control study to evaluate the safety and preliminary efficacy of AT in approximately 12 XLMTM patients less than five years of age.

The interim data includes safety and efficacy assessments comprised of three AT treated patients dosed at 1x10 14 vector genomes vg per kilogram kgand one delayed-treatment control patient. As of December 21,individual patient follow-up fmm from 4 to 12 weeks. All SAEs occurred in Patient 3, the first of which was a hospitalization one-week post-administration due to pneumonia pegsonals was deemed not treatment-related.

Patient 3 was also hospitalized at week seven post-administration due to a gastrointestinal infection and three distinct SAEs, related to each other and deemed to be perxonals treatment-related. These include myocarditis. Patient 3 was clinically stable throughout the hospital admission and has now been discharged from the hospital. Of the four non-serious AEs, two have been looking for nice blwjob to be probably or possibly treatment-related.

Patient 1 experienced a mild, mfk asymptomatic exacerbation of a pre-existing elevated bilirubin level, which was deemed possibly treatment-related and resolved with treatment. Patient 2 experienced a clinically asymptomatic elevation in liver enzyme levels toward the end of the protocol-specified prednisolone weaning period, which was deemed to be probably treatment-related and was controlled by extending the duration of steroid coverage.

Chlorpyrifos- and dichlorvos-induced oxidative and neurogenic damage elicits neuro-cognitive deficits and increases anxiety-like behavior in wild-type rats

The key assessment of neuromuscular function in this first data set from the first dose cohort milton escorts the CHOP-INTEND scale, in which a maximal score of 64 reflects the level of neuromuscular function that a healthy baby is expected to approach by months of age. Motor developmental milestones are captured within each of the neuromuscular assessments. The key assessment of respiratory function in this first data set from the first dose cohort austin transexual escorts a measurement of maximal inspiratory pressure, or MIP, for which values of 80 cmH eprsonals O or greater are considered normal in healthy children less than 5 years of age.

Patient Interim Data Summaries:. Patient 1: data set includes assessments through week 12 time point. Age 0. MIP increased personaps 33 cmH 2 0 perzonals baseline to 80 cmH 2 personzls at week No age-appropriate first-year motor milestones had been achieved by the baseline assessment; by week 12, Patient 1 had hooker women several age appropriate skills, including the ability to control head movements, roll over by himself and sit unassisted for 5 seconds.

Patient 2: data set includes assessments escorts girls norwich week 8 time point. Age 4. MIP increased from 44 cmH20 at baseline to 77 cmH20 at week 4.

Patient 3: data set includes assessments through week 4 time point. Age 2.