Wright State University School of Medicine

None Insights from Planning Committee Member Shirish IngawaleAs we anticipate the upcoming annual PDA Universe of Pre-Filled Syringes and Injection Devices Conference in Phoenix this 22-23 October, we caught up with a member of the planning committee to get a sneak preview. Shirish Ingawale, PhD, is also the Director of Device Development Leadership at Takeda and has two decades of expertise in the research, development, manufacturing, and commercialization of medical devices, drug delivery technologies, and drug/device combination products. Before joining Takeda in May 2023, Shirish worked at Amgen for a decade where he was accountable for the development, launch, and life-cycle management of combination products including reconstitution kits, pre-filled syringes, mechanical autoinjectors, and electro-mechanical autoinjectors and platforms. Shirish holds a BS in Instrumentation Engineering from the University of Mumbai, India, and earned his MS and PhD in Biomedical Engineering from Wright State University, Dayton, Ohio, USA. That more than qualifies him to share his perspectives on the value of this event for biomedical professionals and engineers like himself. He adds, “I am passionate about continuous learning which helps me gain diverse strengths from these experiences in my professional and personal life.”Let’s face it, there are many professional conferences to choose from for inspiration, education, and meeting potential partners. Shirish says the PDA annual conference, now in its 21st year, is unique because it has a global perspective, is tightly focused, and offers a network of experienced, collegial professionals. He shares one of his top reasons to attend is to experience the different expo booths (more than 120 this year) to see products, technologies, and manufacturing equipment that will improve his job. “It’s all under one roof, and we can touch and feel these solutions dedicated to the field of drug delivery and devices.” He says this conference is invaluable to his work. The Value of the PDA Universe of Pre-Filled Syringes and Injection Devices ConferencePDA stages the largest global conference on pre-filled syringes and injection devices bringing together key industry experts to discuss the breakthroughs that are propelling the industry forward. This platform delivers world-class content and knowledge exchanges with peer experts and contributes to enhancing global health within the pharmaceutical and medical device sectors. Before and after the conference, specialized training courses and the Combination Products Workshop are filled with highly interactive, hands-on learning opportunities.Shirish says that the content offered across the conference, training courses, and workshop focuses on fact-based problem solving. “The sessions identify the shared challenges and hurdles collectively faced by the industry. The content also drives alignment on pragmatic solutions to bring therapies to the patients (e.g., it deconstructs the ambiguity or absence of clear regulatory expectations and approaches in certain areas such as human factors, identifying the shared challenges faced by the industry).” The takeaway, he adds, is to “encourage the players in the industry – medical device and biotech/pharma – to share lessons (as much as practically possible) and create avenues for meaningful exchange.”The speakers go beyond theory to tackle practical solutions with actionable learning about new trends, challenges, solutions, and opportunities in the drug delivery space.The agenda is developed by peers for peers and covers critical topics, including sustainability, large-volume injections, digital health, and regulatory compliance. Shirish adds that the conference, training courses, and workshop create a “one-stop-shop for curious and innovative scientists, engineers, business leaders and literally anyone interested in prefilled syringes, drug delivery devices, and combination products.” He adds that he is looking forward to moderating the plenary session on “Drug Delivery Platforms – Compare and Contrast Device and Pharma Perspectives” as well as a concurrent session on “Integrated Approaches in Drug-Device Combination Products” on Wednesday, 23 October, during the conference. Why Attend?Shirish shares that the downside risk of skipping this conference is to “miss the unique opportunity to interact in person with a global community that is the best in the world!” Meeting and working in person with scientists, engineers, and business leaders at the top of their game is essential to career growth and to gain a deeper understanding of key industry issues. The high level of interactivity with poster presentations and networking is essential for every professional to become a more valuable asset to their organizations. He adds that he is “looking forward to presentations and discussions on several key areas of drug delivery devices and combination products – from formulation, container, and devices through development, launch and lifecycle management of combination products…and networking with the experts.”Shirish urges PDA members to get involved. He believes volunteering for planning committees is a meaningful experience and members can make a significant difference in the industry as well as for their peers by shaping the content and strengthening the opportunity to make connections and develop partnerships.As a final note to anyone sitting on the fence about attending, Shirish says, “If you’re interested in learning more about drug delivery technologies and combination products from the best in the business, or simply networking with the key players across the globe, do not miss this event!” Visit pda.org/ups2024 to learn more about the conference, training courses, and workshop.

ALACHUA, Fla. and TAMPA, Fla., June 24, 2024 (GLOBE NEWSWIRE) -- Axogen, Inc. (NASDAQ: AXGN), a global leader in developing and marketing innovative surgical solutions for peripheral nerve injuries, announces the launch of its newest product, Avive+ Soft Tissue Matrix™. Avive+™ is intended for use as a soft tissue barrier and is a resorbable, multi-layer amniotic membrane allograft that provides temporary protection and tissue separation during the critical phase of peripheral nerve healing. Avive+ is processed and distributed in accordance with U.S. Food and Drug Administration (FDA) requirements for Human Cellular and Tissue-based Products (HCT/P) under 21 CFR Part 1271 regulations, U.S. State regulations as a 361 human tissue product. “Product innovation is integral to our mission at Axogen," stated Karen Zaderej, Chairman, CEO, and President. "With the introduction of Avive+ Soft Tissue Matrix, we are excited to enhance our comprehensive solutions for nerve protection and address a significant need for patients with compression or non-transected nerve injuries.” Nerve protection represents approximately $800 million of the overall nerve repair market and covers a wide range of injuries and defects including carpal and cubital tunnel syndromes, crush injuries, and other non-transected traumatic nerve injuries. The diversity of these injury types and their anatomical locations present unique challenges and Axogen is committed to optimizing outcomes for patients, using its targeted portfolio of solutions to address the needs of each injury type, and the healing process. Avive+ Soft Tissue Matrix complements the Company’s existing portfolio of nerve protection products, which includes Axoguard Nerve Protector® and Axoguard HA+ Nerve Protector™. "As a hand surgeon, I've seen the positive impact of using Avive+ Soft Tissue Matrix,” stated Sunishka Wimalawansa, MD, Associate Professor of Orthopedic and Plastic Surgery at Wright State University in Dayton, OH. "The multi-layer design provides ideal handling, a robust barrier that retains the inherent properties of an amniotic membrane, and most importantly, the opportunity to improve patient outcomes after acute traumatic injury." About Axogen Axogen (AXGN) is the leading Company focused specifically on the science, development, and commercialization of technologies for peripheral nerve regeneration and repair. Axogen employees are passionate about helping to restore peripheral nerve function and quality of life to patients with physical damage or transection to peripheral nerves by providing innovative, clinically proven, and economically effective repair solutions for surgeons and health care providers. Peripheral nerves provide the pathways for both motor and sensory signals throughout the body. Every day, people suffer traumatic injuries or undergo surgical procedures that impact the function of their peripheral nerves. Physical damage to a peripheral nerve, or the inability to properly reconnect peripheral nerves, can result in the loss of muscle or organ function, the loss of sensory feeling, or the initiation of pain. Axogen's platform for peripheral nerve repair features a comprehensive portfolio of products used across various applications and surgical specialties, including traumatic injuries, oral and maxillofacial surgery, breast reconstruction, and the surgical treatment of pain. These applications encompass both scheduled and emergent procedures. Specifically, scheduled procedures are often pursued by patients seeking relief from conditions caused by a nerve defect or previous surgical interventions. Such procedures include providing sensation for women undergoing breast reconstruction following a mastectomy, nerve reconstruction after the surgical removal of painful neuromas, and oral and maxillofacial procedures, as well as nerve decompression. Conversely, emergent procedures typically arise from injuries that initially present in an emergency room, with specialists intervening either immediately or within a few days following the initial injury. This broad range of applications underscores Axogen’s vital role in addressing diverse patient needs in peripheral nerve repair. Axogen's platform for peripheral nerve repair features a comprehensive portfolio of products, including Avance® Nerve Graft, a biologically active off-the-shelf processed human nerve allograft for bridging severed peripheral nerves without the comorbidities associated with a second surgical site; Axoguard Nerve Connector®, a porcine submucosa extracellular matrix (ECM) coaptation aid for tensionless repair of severed peripheral nerves; Axoguard Nerve Protector®, a porcine submucosa ECM product used to wrap and protect damaged peripheral nerves and reinforce the nerve reconstruction while preventing soft tissue attachments; Axoguard HA+ Nerve Protector™, a porcine submucosa ECM base layer coated with a proprietary hyaluronate-alginate gel, a next-generation technology designed to enhance nerve gliding and provide short- and long-term protection for peripheral nerve injuries; Avive+ Soft Tissue Matrix™, a multi-layer amniotic membrane allograft used to protect and separate tissues in the surgical bed during the critical phase of tissue repair; and Axoguard Nerve Cap®, a porcine submucosa ECM product used to protect a peripheral nerve end and separate the nerve from the surrounding environment to reduce the development of symptomatic or painful neuroma. The Axogen portfolio of products is available in the United States, Canada, the United Kingdom, South Korea, and several other European and international countries. For more information, visit Cautionary Statements Concerning Forward-Looking Statements This press release contains “forward-looking” statements as defined in the Private Securities Litigation Reform Act of 1995. These statements are based on management's current expectations or predictions of future conditions, events, or results based on various assumptions and management's estimates of trends and economic factors in the markets in which we are active, as well as our business plans. Words such as “expects,” “anticipates,” “intends,” “plans,” “believes,” “seeks,” “estimates,” “projects,” “forecasts,” “continue,” “may,” “should,” “will,” “goals,” and variations of such words and similar expressions are intended to identify such forward-looking statements. Forward-looking statements include Ms. Zaderej’s statement that Avive+ Soft Tissue Matrix will address a significant need for patients with compression or non-transected nerve injuries, as well as statements relating to the estimated size of the nerve protection market. Actual results or events could differ materially from those described in any forward-looking statements as a result of various factors, including, without limitation, global supply chain issues, hospital staffing issues, product development, product potential, clinical outcomes, regulatory process and approvals, financial performance, sales growth, surgeon and product adoption, market awareness of our products, data validation, our visibility at and sponsorship of conferences and educational events, global business disruption caused by Russia’s invasion of Ukraine and related sanctions, recent geopolitical conflicts in the Middle East, potential disruptions due to management transitions, as well as those risk factors described under Part I, Item 1A, “Risk Factors,” of our Annual Report on Form 10-K for the most recently ended fiscal year. Forward-looking statements are not a guarantee of future performance, and actual results may differ materially from those projected. The forward-looking statements are representative only as of the date they are made and, except as required by applicable law, we assume no responsibility to publicly update or revise any forward-looking statements. Contact: Axogen, Inc. Harold D. Tamayo, Vice President of Finance, and Investor Relations htamayo@axogeninc.com

NEW YORK, May 7, 2024 /PRNewswire/ -- Myrtelle Inc. ("Myrtelle" or the "Company"), a clinical-stage gene therapy company focused on developing transformative treatments for neurodegenerative diseases, will host a symposium on Canavan disease (CD) clinical trial updates and future directions for oligodendrocyte targeting AAVs at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting on Wednesday, May 8, 2023, at 8:30 am EDT in Baltimore. The symposium will highlight development of gene therapy targeting oligodendrocytes for Canavan disease, updates on the company's First in Human (FIH) open-label Phase 1/2 clinical trial using its proprietary vector (rAAV-Olig001-ASPA), and the discovery of novel oligodendrocyte targeting AAV capsids. Oligodendrocytes perform numerous key functions in the brain - including the production of myelin, the insulating material that enables proper neuronal function – and are now appreciated as having roles in a range of diseases. Myrtelle is developing a unique toolkit, including a proprietary class of recombinant adeno-associated virus (rAAV) vectors that directly target these cells thereby providing potential treatment options for diseases in the company pipeline. Symposium speakers include: - Paola Leone, Ph.D., Professor and Director of the Cell and Gene Therapy Center at Rowan-Virtua SOM & School of Translational Biomedical Engineering & Sciences Virtua Health College of Medicine and Life Sciences of Rowan University will cover the history of her work in Canavan disease in her talk on the "Development of a Gene Therapy for Canavan Disease Targeting Oligodendrocytes." - Rob Lober, M.D. Ph.D. Principal Investigator on Myrtelle's Canavan Disease Gene Therapy Trial and Attending Neurosurgeon at Dayton Children's Hospital and Associate Professor of Pediatrics at Wright State University Boonshoft School of Medicine will present on a case study from Myrtelle's Phase 1/2 First-in-Human Gene Therapy Clinical Trial in Patients with Canavan Disease in his talk titled, "Oligodendrocyte Targeted Gene Therapy for Canavan Disease." - Patrick Aldrin-Kirk, Ph.D. Chief Science Officer at rAAVen, an AAV engineering company focused on generating innovative vectors for the next generation of gene therapies, will present on the "Discovery of Novel Oligodendrocyte Targeting AAV Capsids" and potential disease targets. "As a leader in oligodendrocyte targeted therapies, we are thrilled to have the opportunity to host a symposium at ASGCT's annual conference," said Jordana Holovach, Head of Communications and Community at Myrtelle. "This symposium provides a unique forum to highlight the critical but underappreciated role of oligodendrocytes in the overall health of the brain and as a potential target for treating neurodegenerative diseases. We are excited by the potential of our evolving toolbox for delivering therapeutic constructs to oligodendrocytes and impacting disease targets that are inherently intractable by nature. We look forward to sharing our insights and generating further interest into the oligodendrocyte story at the symposium." Date: Wednesday, May 8, 2024 Location: Baltimore Convention Center – Room 324 – 326 Time: 8:30 am – 9:30 am ABOUT MYRTELLE Myrtelle Inc. is a gene therapy company focused on developing transformative treatments for neurodegenerative diseases. The company has a proprietary platform, intellectual property, and portfolio of programs and technologies supporting innovative gene therapy approaches for neurodegenerative diseases. Myrtelle has an exclusive worldwide licensing agreement with Pfizer Inc. for its Canavan disease program. For more information, please visit the Company's website at: . ABOUT CANAVAN DISEASE Canavan disease (CD) is a fatal childhood genetic brain disease caused by mutations in the ASPA gene (ASPA) which prevent the normal expression of aspartoacylase, a critical enzyme produced in oligodendrocytes. The lack of normal aspartoacylase expression negatively impacts brain development, including myelin production. Patients with CD are impacted at birth but may appear normal until several months old when symptoms begin to develop. Poor head control, abnormally large head size, difficulty in eye tracking, excessive irritability, severely diminished muscle tone, and delays in reaching motor milestones, such as rolling, sitting, and walking, are the typical initial manifestations of CD. As the disease progresses, seizures, spasticity, difficulties in swallowing, and overall muscle deterioration emerge with most affected children developing life-threatening complications by approximately 10 years of age. Currently, there are no cures for CD, and only palliative treatments are available. More information on Myrtelle's clinical trial in Canavan disease can be found on under the identifier NCT04833907 or by emailing PatientAdvocacy@MyrtelleGTX.com. Forward-Looking Statements This press release contains forward-looking statements. Words such as "may," "believe," "will," "expect," "plan," "anticipate," "estimate," "intend" and similar expressions (as well as other words or expressions referencing future events, conditions, or circumstances) are intended to identify forward-looking statements. Forward-looking statements are based upon current estimates and assumptions and include statements regarding results obtained to date continuing to reinforce the hypothesis that addressing the gene deficit directly in the brain, and specifically in oligodendrocytes, of patients with Canavan disease can potentially restore myelination and support functional improvements. While Myrtelle believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements, which are based on information available to us on the date of this release. These forward-looking statements are subject to various risks and uncertainties, many of which are difficult to predict, that could cause actual results to differ materially from current expectations and assumptions from those set forth or implied by any forward-looking statements. Important factors that could cause actual results to differ materially from current expectations include, among others, Myrtelle's program demonstrating safety and efficacy, as well as future results that are consistent with prior results, the ability to generate the data needed for further development of this novel gene therapy in the patients with CD, and the ability to continue its clinical trials and to complete them on time and achieve the desired results. All forward-looking statements are based on Myrtelle's expectations and assumptions as of the date of this press release. Actual results may differ materially from these forward-looking statements. Except as required by law, Myrtelle expressly disclaims any responsibility to update any forward-looking statement contained herein, whether as a result of new information, future events, or otherwise. View original content to download multimedia: SOURCE Myrtelle, Inc