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Category Archives: Genetic Medicine

NeuBase Therapeutics Appoints Gerald J. McDougall to Board of Directors – BioSpace

PITTSBURGH, May 12, 2021 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology company accelerating the genetic revolution with a new class of precision genetic medicines, announced today the appointment of Gerald (Gerry) J. McDougall to the Company's Board of Directors. For more than 25 years, Mr. McDougall has been the driving force behind large-scale strategic alliances, joint ventures, and industry partnerships across the healthcare industry to advance innovations in precision medicine and cancer.

"Gerry's deep expertise, passionate commitment to improve the human condition, and vast network have been the foundation for numerous transformational alliances in life sciences and healthcare, and his ability to create synergistic combinations of people, ideas, and resources is exceptional," said Dietrich A. Stephan, Ph.D., Founder, CEO and Chairman of NeuBase. "The Board and I look forward to working closely with Gerry as we advance NeuBase's comprehensive approach to precision genetic medicine to address previously untreatable diseases."

"Genetics are the foundation for understanding and treating rare and common diseases including cancers, and the ability to precisely modulate gene function is key to developing new medicines for the many diseases that still have no treatment options," said Mr. McDougall. "I have dedicated my career to coalescing divergent approaches to achieve precision care, and I believe NeuBase can unify the field of precision genetic medicine with its PATrOL technology platform."

Mr. McDougall spent almost his entire career as a senior partner at PwC where he built and led the firm's Global Health Science consulting practice before retiring. He has worked across the entire ecosystem of the healthcare industry and advised an array of Fortune 500 companies, including leading global pharmaceutical companies. Mr. McDougall has been instrumental around the globe in building public-private partnerships to address human health imperatives. These include the creation and maturation of the Translational Genomics Research Institute (TGen), Arizona's renowned bio-cluster; the design and launch of the Multiple Myeloma Research Consortium (MMRC); the strategic plan for the California Institute of Regenerative Medicine (CIRM) and the Country of Luxemburg's biotechnology commercialization ecosystem.

About NeuBase Therapeutics, Inc.NeuBase is accelerating the genetic revolution by developing a new class of precision genetic medicines which can be designed to increase, decrease, or change gene function, as appropriate, to resolve genetic defects that drive disease. NeuBase's targeted PATrOL therapies are centered around its proprietary drug scaffold to address genetic diseases at the DNA or RNA level by combining the highly targeted approach of traditional genetic therapies with the broad organ distribution capabilities of small molecules. With an initial focus on silencing disease-causing mutations in debilitating neurological, neuromuscular, and oncologic disorders, NeuBase is committed to redefining medicine for the millions of patients with both common and rare conditions. To learn more, visit http://www.neubasetherapeutics.com.

Use of Forward-Looking StatementsThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act. These forward-looking statements are distinguished by use of words such as "will," "would," "anticipate," "expect," "believe," "designed," "plan," or "intend," the negative of these terms, and similar references to future periods. Forward-looking statements include, among others, those related to the anticipated strategic guidance and assistance that the Company's new director will provide to support the Company's comprehensive approach to precision genetic medicine to address previously untreatable diseases. These views involve risks and uncertainties that are difficult to predict and, accordingly, our actual results may differ materially from the results discussed in our forward-looking statements. Our forward-looking statements contained herein speak only as of the date of this press release. Factors or events that we cannot predict, including those risk factors contained in our filings with the U.S. Securities and Exchange Commission (the SEC), may cause our actual results to differ from those expressed in forward-looking statements. The Company may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements, and you should not place undue reliance on these forward-looking statements. Because such statements deal with future events and are based on the Company's current expectations, they are subject to various risks and uncertainties, and actual results, performance or achievements of the Company could differ materially from those described in or implied by the statements in this press release, including: the Company's plans to develop and commercialize its product candidates; the timing of initiation of the Company's planned clinical trials; the timing of the availability of data from the Company's clinical trials; the timing of any planned investigational new drug application or new drug application; the Company's plans to research, develop and commercialize its current and future product candidates; the clinical utility, potential benefits and market acceptance of the Company's product candidates; the Company's commercialization, marketing and manufacturing capabilities and strategy; global health conditions, including the impact of COVID-19; the Company's ability to protect its intellectual property position; and the requirement for additional capital to continue to advance these product candidates, which may not be available on favorable terms or at all, as well as those risk factors contained in our filings with the SEC. Except as otherwise required by law, the Company disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.

NeuBase Investor Contact:Dan FerryManaging DirectorLifeSci Advisors, LLCdaniel@lifesciadvisors.comOP: (617) 430-7576

NeuBase Media Contact:Jessica Yingling, Ph.D.Little Dog Communications Inc.jessica@litldog.com+1 (858) 344-8091

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NeuBase Therapeutics Appoints Gerald J. McDougall to Board of Directors - BioSpace

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NeuBase Therapeutics Reports Financial Results for the Second Quarter of Fiscal Year 2021 – GlobeNewswire

PITTSBURGH, May 13, 2021 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology company accelerating the genetic revolution using a new class of precision genetic medicines, today reported its financial results for the three- and six-month periods ended March 31, 2021.

"We continue to expand and scale our unique precision genetic medicine platform that we believe can turn genes on, off, or edit them in vivo, and thus address most mechanisms that cause diseases in a single industry-unifying solution, said Dietrich A. Stephan, Ph.D., Founder, CEO and Chairman of NeuBase. Our recent financing led by top-tier healthcare investors enables us to advance our lead program into the clinic next year and expand our pipeline to address historically undruggable oncogenic driver mutations. We look forward to hosting our R&D day on June 8th, during which we will present an update on our current pipeline programs, as well as introduce an oncology program targeting a genetic driver mutation in a high value indication.

Second Quarter of Fiscal Year 2021 and Recent Operating Highlights

Financial Results for the Second Fiscal Quarter Ended March 31, 2021

Financial Results for the Six-Month Period Ended March 31, 2021

About NeuBase TherapeuticsNeuBase is accelerating the genetic revolution by developing a new class of precision genetic medicines which can be designed to increase, decrease, or change gene function, as appropriate, to resolve genetic defects that drive disease. NeuBases targeted PATrOL therapies are centered around its proprietary drug scaffold to address genetic diseases at the DNA or RNA level by combining the highly targeted approach of traditional genetic therapies with the broad organ distribution capabilities of small molecules. With an initial focus on silencing disease-causing mutations in debilitating neurological, neuromuscular and oncologic disorders, NeuBase is committed to redefining medicine for the millions of patients with both common and rare conditions. To learn more, visit http://www.neubasetherapeutics.com.

Use of Forward-Looking StatementsThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act. These forward-looking statements are distinguished by use of words such as "will," "would," "anticipate," "expect," "believe," "designed," "plan," or "intend," the negative of these terms, and similar references to future periods. These forward-looking statements include, among others, those related to the potential significance and implications of the Company's positive in vitro and in vivo preclinical data for its PATrOL-enabled anti-gene therapies for the treatment of myotonic dystrophy type 1 (DM1), the plan to provide updates on the Company's development pipeline, including the myotonic dystrophy type 1 (DM1) and Huntington's disease (HD) programs and an oncology program targeting a genetic driver mutation in a high value indication, at an R&D day in June 2021, the anticipated use of proceeds from the Companys April 2021 public equity offering and the Company's anticipated capital requirements over approximately the next twelve months. These views involve risks and uncertainties that are difficult to predict and, accordingly, our actual results may differ materially from the results discussed in our forward-looking statements. Our forward-looking statements contained herein speak only as of the date of this press release. Factors or events that we cannot predict, including those risk factors contained in our filings with the U.S. Securities and Exchange Commission (the SEC), may cause our actual results to differ from those expressed in forward-looking statements. The Company may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements, and you should not place undue reliance on these forward-looking statements. Because such statements deal with future events and are based on the Company's current expectations, they are subject to various risks and uncertainties, and actual results, performance or achievements of the Company could differ materially from those described in or implied by the statements in this press release, including: the Company's plans to develop and commercialize its product candidates; the timing of initiation of the Company's planned clinical trials; the risks that prior data will not be replicated in future studies; the timing of any planned investigational new drug application or new drug application; the Company's plans to research, develop and commercialize its current and future product candidates; the clinical utility, potential benefits and market acceptance of the Company's product candidates; the Company's commercialization, marketing and manufacturing capabilities and strategy; global health conditions, including the impact of COVID-19; the Company's ability to protect its intellectual property position; and the requirement for additional capital to continue to advance these product candidates, which may not be available on favorable terms or at all, as well as those risk factors contained in our filings with the SEC. Except as otherwise required by law, the Company disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.

NeuBase Investor Contact:Dan FerryManaging DirectorLifeSci Advisors, LLCdaniel@lifesciadvisors.com OP: (617) 430-7576

NeuBase Media Contact:Jessica Yingling, Ph.D.Little Dog Communications Inc.(858) 344-8091jessica@litldog.com

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NeuBase Therapeutics Reports Financial Results for the Second Quarter of Fiscal Year 2021 - GlobeNewswire

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Biogen looks to build better gene therapies through latest deal – BioPharma Dive

Dive Brief:

Biogen's big move into gene therapy came just over two years ago with the $800 million acquisition of Nightstar Therapeutics, a developer of eye medicines.

Since then, the company has inked an agreement with Sangamo Therapeutics to explore gene editing as a way to treat neurological diseases, pushed further into gene therapies for the eye through a deal with Germany's ViGeneron, and just recently announced plans to spend $200 million on a gene therapy manufacturing facility in North Carolina.

These investments have come both as Biogen's peers pour money into gene therapy, and as the company faces investor pressure to diversify its business.

Gene therapy research has made a resurgence, in large part, because the technology and how it interacts with the body are now better understood. The Food and Drug Administration has approved a handful of cell and gene therapies over the last few years, and expects to be reviewing and approving between 10 and 20 annually by 2025.

Yet, there are still major challenges in this cutting-edge field, with one of the biggest being delivery. To be effective, gene therapies must achieve the complicated task of carrying their payloads to the appropriate cells without getting destroyed by the body's defense mechanisms. That task can be especially difficult for therapies going after highly protected areas, such as the brain.

Companies that specialize in gene therapy delivery have therefore been in high demand. Over the last year, for example, a startup called Dyno Therapeutics has caught the attention of heavyweight gene therapy developers like Novartis, Roche and Sarepta Therapeutics.

Dyno's capsid technology is meant to create more targeted gene therapies that are less likely to trigger an immune response. Just last week, the company said it had raised an additional $100 million in fresh funding from tech investor Andreessen Horowitz and several other venture firms.

Also recently, the gene editing startup Beam Therapeutics agreed to buy Guide Therapeutics, which makes tools to deliver genetic medicines into cells, in a deal valued at $120 million.

Capsigen now adds to this flurry of activity with its Biogen collaboration. Per deal terms, Biogen will hold an exclusive license to Capsigen's technology for an undisclosed number of CNS and neuromuscular disease targets.

"One of our priorities for technology innovation is the discovery of AAV capsids with improved delivery profiles," Al Sandrock, Biogen's head of research and development, said in a May 11 statement. "We are investing for the long-term by building platform capabilities and advanced manufacturing technologies with the goal of accelerating our efforts in gene therapy."

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Biogen looks to build better gene therapies through latest deal - BioPharma Dive

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Gene and Cell Therapy Breakthroughs Focus of World Medical – GlobeNewswire

Boston, MA, May 13, 2021 (GLOBE NEWSWIRE) -- Gene and cell therapy (GCT), a transformative approach to develop treatments, and, potentially, cures for congenital and other diseases, is the focus of this years World Medical Innovation Forum May 19-21. Widely recognized as a transformational opportunity in medicine, GCT has the potential to stop or slow the effects of disease by targeting them at the genetic level. When the genetic driver for a disease is known, patients can be molecularly matched to therapies.

Featuring a unique combination of nearly 200 speakers and panelists from Mass General Brighams Harvard-affiliated faculty, industry, venture, manufacturing, and regulatory agencies, the World Medical Innovation Forum, in an all-virtual format, will explore interdependent aspects of advancing GCT for patients worldwide. Topics cover GCT strategy, clinical opportunities, patient access, economic and investment considerations, regulatory frameworks and manufacturing scalability. Nearly 1000 organizations will be represented, including attendees from 43 countries and 40 states of the U.S.

The Forum includes updates on:

GCT Potential for PatientsGene Therapies for Specific Diseases Large Incidence and Rare Trends in Vector Development, including AAV, Lentivirus and Nano MethodsClinical Opportunities, Technology and Market PotentialRegulatory Frameworks for GCTShaping GCT InnovationCancer, Gene Therapy and Oncolytic VirusesCAR-T TherapyGCT Manufacturing, Supply Chain and ScalabilityImpact of mRNA Vaccines

The Forum fosters critical discussions and debates on issues that will determine the pace of expansion of GCT therapies for patients in the coming years, says Chris Coburn, Chief Innovation Officer, Mass General Brigham. In the spirit of collaborative innovation, this gathering of leading investors, entrepreneurs, industry executives, Harvard clinicians and scientists is unique in convening top decision-makers in sharing ideas for advancing these game-changing technologies to the front lines of medicine.

We are honored to be the presenting sponsor of this years World Medical Innovation Forum, said Dave Lennon, President, Novartis Gene Therapies. Having brought one of the first gene therapies to market, we have seen the enormous potential these breakthrough medicines hold to transform lives. We look forward to joining other leaders at the forum to share learnings and our vision for the future one in which gene therapies drive scientific innovation and deliver on their promise for patients and societies.

Showcasing Mass General Brigham BreakthroughsThe Forum showcases GCT innovation breakthroughs from Mass General Brighams Harvard Medical School faculty investigators in 10-minute First Look presentations, and a research poster session being included in the Forum for the first time. More than 40 emerging GCT research projects will be part of the virtual showcase, with topics including gene therapy for large incidence and rare diseases including neurology, cardiology, blood disorders, and oncology, CAR-T breakthroughs, and oncolytic, AAV and non-viral delivery methods.

Recipients of Mass General Brighams Innovation Discovery Grants for GCT research will be announced. The Forum concludes with the announcement of the annual Disruptive Dozen, 12 GCT advances members of the Mass General Brigham faculty believe are the most likely to break through in the next few years.

This years Forum co-chairs are Nino Chiocca, MD, PhD, Chair, Neurosurgery, Brigham and Womens Hospital and Cushing Professor of Neurosurgery, Harvard Medical School (HMS); Sue Slaugenhaupt, PhD, Scientific Director and Elizabeth G. Riley and Daniel E. Smith Jr., Endowed Chair, Mass General Research Institute, and Professor, Neurology, HMS; Ravi Thadhani, MD, CAO, Mass General Brigham and Professor, Medicine and Faculty Dean, HMS; and Luk Vandenberghe, PhD, Grousbeck Family Chair, Gene Therapy, Mass Eye and Ear, and Associate Professor, Ophthalmology, HMS.

About the World Medical Innovation Forum

The World Medical Innovation Forum was established seven years ago in response to the intensifying transformation of health care and its impact on innovation. The Forum is rooted in the belief that no matter the magnitude of change, the center of health care needs to be a shared, fundamental commitment to collaborative innovation industry and academia working together to improve patient lives.

To find out more about the Forum, speakers and agenda, and to register, visit https://worldmedicalinnovation.org/###

About Mass General Brigham

Mass General Brigham is an integrated academic healthcare system, uniting great minds in medicine to make life-changing impact for patients in our communities and people around the world. Mass General Brigham connects a full continuum of care across a system of academic medical centers, community and specialty hospitals, a health insurance plan, physician networks, community health centers, home care, and long-term care services. Mass General Brigham is a non-profit organization that is committed to patient care, research, teaching, and service to the community. In addition, Mass General Brigham is one of the nations leading biomedical research organizations and a principal teaching affiliate of Harvard Medical School. For more information, please visit massgeneralbrigham.org.

About Mass General Brigham Innovation

Innovation is the 150-person business development unit of Mass General Brigham responsible for the worldwide commercial application of the unique capabilities and discoveries of Mass General Brigham's 74,000 employees. Innovation supports the research requirements of its 6,200 Harvard Medical School faculty and research hospitals. It has responsibility for industry collaborations, venture investing, international consulting, licensing, innovation management, company creation, technology marketing, open innovation alliances, and workforce development.

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Gene and Cell Therapy Breakthroughs Focus of World Medical - GlobeNewswire

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From one genomic diagnosis, researchers discover other treatable health conditions – National Human Genome Research Institute

Genome sequencing the ability to sequence an individual's DNA is becoming a standard tool to study diseases. In 2019, over 26 million people took direct-to-consumer DNA tests, which speaks to our collective desire to better understand our genomes.

In July 2013, the American College of Medical Genetics and Genomics issued a recommendation that people who have their genomes sequenced in a clinical setting should also have their genomic data screened for variants in 56 genes that can pose health risks. The genes (which includes the RET gene) are associated with increased risks for several life-threatening, but treatable or preventable diseases. The number of genes included in the list increased to 59 in 2016, and clinicians expect that the list will be updated again soon.

When a person comes into the clinic to be tested for a specific condition, any positive result related to that condition is called a primary finding. But when testing reveals information separate from the original condition, it is called a secondary finding. An estimated 1-4% of people receive unexpected health results from genomic tests each year.

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Animation of a clinician explaining primary and secondary findings. Credit: Harry Wedel, NHGRI.

Secondary findings are not unique to clinical genomics. People can also receive secondary findings from MRI, radiology and other medical tests.

In the case of genome sequencing, examples of secondary findings can include those related to the BRCA1 and BRCA2 genes, which are associated with breast and ovarian cancer risk, and conditions such as inherited heart rhythm problems. Clinicians share such secondary findings with a person only if the person consents to receiving such information.

Secondary findings are now a component of precision medicine, relying on individual and collective genomic data to make assessments about a persons health risks. Clinicians can obtain highly accurate findings because of the vast amount of available genome sequence data. Researchers can search this data to improve genomic testing and how they detect people who are at risk of harboring disease-related variants. Specifically, secondary findings provide individuals and families the opportunity to learn about their health risks before they develop a disease.

In 2019, researchers at the National Human Genome Research Institute (NHGRI) started the Genomic Services Research Program, part of what is now NHGRI's Center for Precision Health Research, to further understand and improve the implementation of precision medicine initiatives.

"Secondary findings play a pivotal role in diagnosing diseases, preserving health and saving lives," said Leslie Biesecker, M.D., chief of the NHGRIs Clinical Genomics Section. "Our research program measures how clinicians communicate these findings and peoples reactions so we can identify areas for improvement. The payoff could improve human health by making it commonplace for people to get treatment for diseases before they are sick."

The payoff could improve human health by making it commonplace for people to get treatment for diseases before they are sick.

According to Biesecker, identifying a secondary finding is only the first step. The Genomic Services Research Program studies whether secondary findings have real-life use and value by assessing three key components:

Biesecker also emphasized the need for healthcare providers to clearly communicate with patients who receive secondary findings so they understand their treatment options.

"Most people seek out genetic testing because they know of a strong family history for a certain disease," said Katie Lewis, Sc.M., CGC, a genetic counselor in the program. "But for those individuals who get these secondary findings, it can be an immense surprise. Our goal is to help individual patients get the care they need and share the result with their families.

Lewis also adds that there is very little known about the extent to which patients follow through with treatment and the factors that influence their decisions. Understanding what motivates those who do take action and those who do not can help genetic counselors target their efforts to assist an individual with a secondary finding and translate it into improved long-term health.

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From one genomic diagnosis, researchers discover other treatable health conditions - National Human Genome Research Institute

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Akouos Presents Nonclinical Data Supporting Future Clinical Development of AK-OTOF and AK-antiVEGF at the American Society of Gene and Cell Therapy…

- Intracochlear delivery of a dual AAVAnc80 vector encoding human otoferlin results in full-length protein expression in inner hair cells of non-human primates and in durable protein expression sufficient for sustained restoration of auditory function in Otof knockout mice

- Multiple analyses demonstrate in vitro transduction with dual AK-OTOF vector results in full-length otoferlin expression, with no detection of truncated proteins

- Long-term, local expression of anti-VEGF protein is robust and well tolerated following intracochlear administration of AK-antiVEGF in non-human primates

- Akouos continues to progress towards planned IND submissions for AK-OTOF in the first half of 2022 and for AK-antiVEGF in 2022

BOSTON, May 11, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ: AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today presented nonclinical data supporting the future clinical development of both AK-OTOF, a gene therapy intended for the treatment of otoferlin gene (OTOF)-mediated hearing loss, and AK-antiVEGF, a gene therapy intended for the treatment of vestibular schwannoma, in three digital presentation sessions at the virtual American Society of Gene and Cell Therapy (ASGCT) 24th Annual Meeting.

We are excited to share new data that highlight the potential of genetic medicines for inner ear conditions with the broader gene therapy community, said Manny Simons, Ph.D., M.B.A., co-founder, president, and chief executive officer of Akouos. Inner ear conditions represent one of the largest areas of unmet need in medicine today, and one of the challenges in this area is the ability to efficiently address the broad range of conditions that collectively affect hundreds of millions of individuals worldwide. The nonclinical data presented today for the AK-OTOF and AK-antiVEGF programs demonstrate how we are leveraging our genetic medicines platform and multiple AAV-mediated modalities, including gene transfer and therapeutic protein expression, to begin to address that challenge.

Nonclinical data presented at ASGCT for AK-OTOF continue to support the potential to restore physiologic hearing and provide long-lasting benefit to individuals with OTOF-mediated hearing loss. In Otof knockout mice, AK-OTOF administration results in durable expression of human otoferlin protein sufficient for sustained restoration of auditory function. In addition, data presented indicate that expression of exogenous secreted protein at or above reported biologically active levels, driven by a ubiquitous promoter, is well tolerated in non-human primates following administration of AK-antiVEGF. These IND-enabling nonclinical studies are promising and support future clinical development. Our team continues to work towards submission of INDs for AK-OTOF and AK-antiVEGF expected in 2022, said Greg Robinson, Ph.D., chief scientific officer of Akouos.

In Vitro and In Vivo Analyses of Dual Vector Otoferlin Expression to Support the Clinical Development of AK-OTOF (AAVAnc80-hOTOF Vector)

Presenting Author: Eva Andres-Mateos

Abstract Number: 355

Otoferlin plays a critical role in exocytosis of synaptic vesicles at the inner hair cell synapse, and mutations inOTOF, the gene encoding otoferlin, are associated with autosomal recessive sensorineural hearing loss. AK-OTOF is designed to deliver normal OTOF by utilizing a dual vector approach,which encodes the 5 and the 3 components of OTOF. Multiple analyses demonstrate in vitro transduction with dual AK-OTOF vector results in full-length human otoferlin (RNA and protein), with no detection of truncated proteins from either AK-OTOF or its component vectors (5hOTOF and 3hOTOF). A one-to-one ratio of the AK-OTOF component vectors appears to be optimal for efficient reconstitution of full-length human otoferlin. In cynomolgus macaques, full-length human otoferlin protein expression is detected in inner hair cells of non-human primate (NHP) cochleae by both immunohistochemistry and immunodetection one month following intracochlear administration of AAVAnc80-FLAG.hOTOF.

The digital presentation is located at https://akouos.com/gene-therapy-resources/.

Durable Recovery of Auditory Function Following Intracochlear Delivery of AK-OTOF (AAVAnc80-hOTOF Vector) in a Translationally Relevant Mouse Model of Otoferlin Gene (OTOF)-Mediated Hearing Loss

Presenting Author: Ann Hickox

Abstract Number: 569

Otoferlin gene (OTOF)-mediated hearing loss is caused by mutations in the OTOF gene and is typically characterized by a congenital, Severe to Profound sensorineural hearing loss. The physiologic deficiency resulting from OTOF mutations is localized; specifically, synaptic transmission between the inner hair cell and the auditory nerve is affected, as measured by an absent or abnormal auditory brain stem response (ABR). Gene therapy for OTOF-mediated hearing loss is expected to confer the greatest benefit when cochlear integrity is preserved, as represented by present otoacoustic emissions (OAEs). Individuals with OTOF-mediated hearing loss typically experience a decline in cochlear integrity within the first decade of life, indicated by initially present, then absent, OAEs. In an Otof knockout mouse model that recapitulates the human phenotype, administration of AK-OTOF, an adeno-associated viral gene therapy vector encoding human otoferlin under the control of a ubiquitous promoter, results in durable restoration of auditory function, as measured by ABRs, and may preserve OAEs.

The digital presentation is located at https://akouos.com/gene-therapy-resources/.

Demonstration of Tolerability of a Novel Delivery Approach and Secreted Protein Expression Following Intracochlear Delivery of AK-antiVEGF (AAVAnc80-antiVEGF Vector) in Non-Human Primates

Presenting Author: John Connelly

Abstract Number: 358

Data published from previous clinical trialsshow that systemic VEGF inhibitor therapy can reduce vestibular schwannoma (VS) tumor volume and improve hearing in some participants with mutations in the NF2 gene. However, toxicity limits the potential of this systemic delivery approach from being a viable treatment option for vestibular schwannoma. The exposure and tolerability of local expression of anti-VEGF protein following bilateral, intracochlear administration of AK-antiVEGF was evaluated through analyses of protein levels, as well as physiologic and histologic evaluations, in NHPs. Long-term, local expression of anti-VEGF protein, driven by a ubiquitous promoter, is robust and well tolerated in NHPs following intracochlear administration of AK-antiVEGF. Computational modelling supports the potential for diffusion of anti-VEGF protein at or above reported biologically active levels to the site of the VS tumor.

The digital presentation is located at https://akouos.com/gene-therapy-resources/.

About Akouos

Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

Cautionary Note Regarding Forward-Looking Statements

Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation, plans, and timing of our future clinical trials and our research and development programs, the timing of our IND submissions for AK-OTOF and AK-antiVEGF, our expectations regarding our manufacturing capabilities and timelines, and the period over which we believe that our existing cash, cash equivalents and marketable securities will be sufficient to fund our operating expenses. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, should, target, will, would, and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: our limited operating history; uncertainties inherent in the development of product candidates, including the initiation and completion of nonclinical studies and clinical trials; whether results from nonclinical studies will be predictive of results or success of clinical trials; the timing of and our ability to submit applications for, and obtain and maintain regulatory approvals for, our product candidates; our expectations regarding our regulatory strategy; our ability to fund our operating expenses and capital expenditure requirements with our cash, cash equivalents, and marketable securities; the potential advantages of our product candidates; the rate and degree of market acceptance and clinical utility of our product candidates; our estimates regarding the potential addressable patient population for our product candidates; our commercialization, marketing, and manufacturing capabilities and strategy; our ability to obtain and maintain intellectual property protection for our product candidates; our ability to identify additional products, product candidates, or technologies with significant commercial potential that are consistent with our commercial objectives; the impact of government laws and regulations; risks related to competitive programs; the potential that our internal manufacturing capabilities and/or external manufacturing supply may experience delays; the impact of the COVID-19 pandemic on our business, results of operations, and financial condition; our ability to maintain and establish collaborations or obtain additional funding; and other factors discussed in the Risk Factors included in the Companys Annual Report on Form 10-K for the year ended December 31, 2020 filed with the Securities and Exchange Commission, and in other filings that the Company makes with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

Contacts

Media:Katie Engleman, 1ABkatie@1abmedia.com

Investors:Courtney Turiano, Stern Investor RelationsCourtney.Turiano@sternir.com

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Akouos Presents Nonclinical Data Supporting Future Clinical Development of AK-OTOF and AK-antiVEGF at the American Society of Gene and Cell Therapy...

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