November 2021

ROCKVILLE, Md., Nov. 9, 2021 /PRNewswire/ — REGENXBIO Inc. (Nasdaq: RGNX) today announced the closing of its Collaboration and License Agreement with AbbVie to develop and commercialize RGX-314, a potential one-time gene therapy for the treatment of wet age-related macular degeneration (wet AMD), diabetic retinopathy (DR) and other chronic retinal diseases. As previously announced on September 13, 2021, under the terms of the agreement, REGENXBIO will receive an upfront payment from AbbVie of $370 million with the potential for REGENXBIO to receive up to $1.38 billion in additional development, regulatory and commercial milestones.

Under the collaboration, REGENXBIO will be responsible for completion of the ongoing trials of RGX-314. AbbVie and REGENXBIO will collaborate and share costs on additional trials of RGX-314, including the planned second pivotal trial evaluating subretinal delivery for the treatment of wet AMD and future trials. AbbVie will lead the clinical development and commercialization of RGX-314 globally. REGENXBIO will participate in U.S. commercialization efforts as provided under a mutually agreed upon commercialization plan.

REGENXBIO and AbbVie will share equally in profits from net sales of RGX-314 in the U.S. and AbbVie will pay REGENXBIO tiered royalties on net sales of RGX-314 outside the U.S. In addition, REGENXBIO will lead the manufacturing of RGX-314 for clinical development and U.S. commercial supply, and AbbVie will lead manufacturing of RGX-314 for commercial supply outside the U.S.

About RGX-314

RGX-314 is being investigated as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. RGX-314 consists of the NAV AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina.

REGENXBIO is advancing research in two separate routes of administration of RGX-314 to the eye, through a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. REGENXBIO has licensed certain exclusive rights to the SCS Microinjector® from Clearside Biomedical, Inc. to deliver gene therapy treatments to the suprachoroidal space of the eye.

About REGENXBIO Inc.

REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO’s NAV® Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

SCS Microinjector® is a trademark of Clearside Biomedical, Inc. All other trademarks referenced herein are registered trademarks of REGENXBIO.

Forward-Looking Statements

This press release includes “forward-looking statements,” within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as “believe,” “may,” “will,” “estimate,” “continue,” “anticipate,” “assume,” “design,” “intend,” “expect,” “could,” “plan,” “potential,” “predict,” “seek,” “should,” “would” or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO’s collaboration with AbbVie and REGENXBIO’s future operations and clinical trials. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO’s expectations and predictions is subject to a number of risks and uncertainties, including the outcome of REGENXBIO’s collaboration with AbbVie and other factors, many of which are beyond the control of REGENXBIO. Refer to the “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” sections of REGENXBIO’s Annual Report on Form 10-K for the year ended December 31, 2020 and comparable “risk factors” sections of REGENXBIO’s Quarterly Reports on Form 10-Q and other filings, which have been filed with the U.S. Securities and Exchange Commission (SEC) and are available on the SEC’s website at www.sec.gov. All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. Except as required by law, REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

Contacts:

Tricia Truehart

Investor Relations and Corporate Communications

347-926-7709

ttruehart@regenxbio.com

Investors:

Brendan Burns, 212-600-1902

brendan@argotpartners.com

Media:

David Rosen, 212-600-1902

david.rosen@argotpartners.com

Study results demonstrate first small molecule approach to significantly reduce phosphorylated and aggregated -synuclein and increase GCase protein levels with transport to the lysosomes in iPSC dopaminergic neurons

Results presented at Michael J. Fox Foundation’s Therapeutic Development Webinar

BETHESDA, Md, Nov. 10, 2021 (GLOBE NEWSWIRE) — Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a biotechnology company directly addressing the urgent need for effective neurodegenerative therapies, today announced data from the Company’s study conducted at the University of Maryland School of Medicine (UMSOM). The study, evaluating two lead Structurally Targeted Allosteric Regulators (STARs) compounds – GT-02287 and GT-02329 – for the treatment of Gaucher and GBA1 Parkinson’s Disease, demonstrated highly statistically significant effects on all tested phenotypes representing a key breakthrough in the potential to treat neurodegenerative disorders characterized by misfolded proteins including Parkinson’s Disease, Alzheimer’s, Gaucher’s Disease and Lewy Body Dementia. Study results were presented at The Michael J. Fox Foundation for Parkinson’s Research’s Innovating from Drug Discover to the Clinic: Novel Approaches to PD Therapeutic Development webinar.

Ricardo Feldman, Ph.D., Associate Professor of Microbiology and Immunology at the University of Maryland School of Medicine, stated, “Our laboratory is using human induced pluripotent stem cells (iPSC) derived from patients with Gaucher and GBA-associated Parkinson’s Disease to test the efficacy of the two lead STAR chaperones developed by Gain Therapeutics. Our studies in iPSC-derived cortical and dopaminergic neurons from neuronopathic Gaucher Disease patients show that these compounds significantly increase the levels of GCase protein, its transport to the lysosome, and its enzymatic activity. In dopaminergic neurons, the two lead STAR chaperones also decrease the levels of the hallmark of Parkinson’s Disease – α-synuclein-p129, demonstrating their potential to treat GBA1-associated Parkinson’s Disease.”

“This breakthrough in the ability to restore enzyme function for Parkinson’s Disease and Gaucher’s Disease is unprecedented for a small molecule and will contribute to a major shift in how we think about treating various neurodegenerative and lysosomal storage diseases with a genetic predisposition,” commented Gain CEO, Eric I. Richman. “This brings tremendous hope to the population of individuals who are diagnosed with various forms of alpha synucleinopathies including those with Parkinson’s Disease, Alzheimer’s, Lewy Body Dementia, and all forms of Gaucher’s Disease. We anticipate initiating IND-enabling studies for Gaucher and Parkinson’s Disease shortly.”

Through its proprietary SEE-Tx® platform, Gain has identified two lead STAR candidates (GT-02287 and GT-02329) which have the ability to restore enzyme function of GCase – an enzyme involved in various neurodegenerative diseases. The compounds characterized in various assays and showed the potential to halt the progression in Parkinson’s patients with GBA1 gene mutations as well as patients whose glucocerebrosidase (GCase) protein is misfolded due to ageing cellular processes. This previous work was supported through grants from The Michael J. Fox Foundation (MJFF) and the Silverstein Foundation for Parkinson’s with GBA.

The study conducted in collaboration with Ricardo A. Feldman, Ph.D., Associate Professor of Microbiology and Immunology at UMSOM and lead investigator, is using human induced pluripotent stem cells (iPSC) for Gaucher and Parkinson’s Disease modeling and drug discovery. Gain’s candidate molecules GT-02287 and GT-02329 have been initially assessed in this unique “disease-in-a-dish” iPSC model derived from Gaucher type 2 and 3 patient cells.

The data generated from the collaboration with University of Maryland School of Medicine in the iPSC models for GBA-associated Parkinson’s Disease and Gaucher Disease showed the following results:

Gaucher Type III dopaminergic neurons

GT-02329 and GT-02287 increased GCase enzyme levels by 129% (p ≤0.001) and 118% (p ≤0.001) respectively in Gaucher type III dopaminergic neurons when compared with untreated cells. GT-02329 and GT-02287 demonstrated an increase in GCase and lysosome co-localisation (p ≤0.05) in Gaucher Type III dopaminergic neurons when compared with untreated cells. GT-02287 showed a marked decrease in phosphorylated α-synuclein-p129 of 41% (p ≤0.0001) and a reduction in aggregated α-synuclein-p129 of 47% (p ≤0.01) in Gaucher Type III dopaminergic neurons when compared with untreated cells. Gaucher Type II dopaminergic neurons: phospho- α -synuclein and aggregated synuclein

GT-02287 reduced phosphorylated α-synuclein-p129 by of 39% at 20μM vs non-treated (Stat. significant: p ≤0.001) GT-02287: Reduction of aggregated α-synuclein-p129 by 64% 20μM vs non-treated (Stat. significant: p ≤0.0001) Gaucher Type III cortical neurons: GlcCer

GT-02329 and GT-02287 reduced the amount of the toxic substrate Glucosylceramide (GlcCer) by 42.9% (p ≤0.05) and by 50.2% ((p ≤0.01) respectively when compared with untreated cells. Gaucher Type II dopaminergic neurons

GT-02329 and GT-02287 also demonstrated increased co-localisation of GCase and lysosomes (p ≤0.05) and (p ≤0.0001) when compared with untreated cells. “We appreciate the close collaboration with Dr. Feldman and the University of Maryland School of Medicine in this important area of research. This data demonstrates that our STARs are able to guide misfolded forms of the GCase enzyme to their proper shape and restore enzymatic activity, establishing an important novel approach for direct treatment of GBA1 Parkinson’s Disease and other alpha-synucleinopathies,” said Manolo Bellotto, Ph.D., General Manager at Gain. “This is a further validation of our platform technology and a promising potential therapeutic opportunity for patients suffering from these debilitating diseases.”

The combined data from Gain Therapeutics and Dr. Feldman’s laboratory suggest that STARs bind to novel allosteric binding sites identified using Gain’s proprietary SEE-Tx drug discovery platform, and stabilize and restore the function of misfolded GCase. In these studies, the restoration of GCase function resulted in the depletion of the toxic phosphorylated and aggregated forms of α-synuclein and a reversal of the neurodegenerative process resulting in improved locomotor activity in Parkinson’s Disease model in rats. Given that STARs are small molecule therapies, they have significant advantages over the current standard of care for disorders associated with protein misfolding, including most enzyme replacement therapies and gene therapies. They can be administered as a simple oral pill and can reach organs and tissues not typically accessible through current therapeutic options, including the brain, bone and cartilage. In addition, the Company believes that STARs are relatively easy and cost-effective to manufacture.

“We are encouraged by these study results of impact on underlying Parkinson’s pathology, a key step toward clinical testing and continued development,” said Marco Baptista, PhD, MJFF Vice President of Research Programs. “The greatest unmet need of people with Parkinson’s remains a treatment to slow or stop disease progression. We are proud to work with innovative companies such as Gain Therapeutics that are driving toward that goal.”

About Gain Therapeutics, Inc. Gain Therapeutics, Inc. is positioned at the confluence of technology and healthcare and focused on redefining drug discovery with its SEE-Tx® target identification platform. By identifying and optimizing allosteric binding sites that have never before been targeted, Gain is unlocking new treatment options for difficult-to-treat disorders characterized by protein misfolding. Gain was established in 2017 with the support of its founders and institutional investors. It has been awarded funding support from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and The Silverstein Foundation for Parkinson’s with GBA, as well as from the Eurostars-2 joint program with co-funding from the European Union Horizon 2020 research and Innosuisse.

For more information, please visit https://www.gaintherapeutics.com

About the University of Maryland School of Medicine Now in its third century, the University of Maryland School of Medicine was chartered in 1807 as the first public medical school in the United States. It continues today as one of the fastest growing, top-tier biomedical research enterprises in the world — with 46 academic departments, centers, institutes, and programs, and a faculty of more than 3,000 physicians, scientists, and allied health professionals, including members of the National Academy of Medicine and the National Academy of Sciences, and a distinguished two-time winner of the Albert E. Lasker Award in Medical Research. With an operating budget of more than $1.2 billion, the School of Medicine works closely in partnership with the University of Maryland Medical Center and Medical System to provide research-intensive, academic and clinically based care for nearly 2 million patients each year. The School of Medicine has nearly $600 million in extramural funding, with most of its academic departments highly ranked among all medical schools in the nation in research funding. As one of the seven professional schools that make up the University of Maryland, Baltimore campus, the School of Medicine has a total population of nearly 9,000 faculty and staff, including 2,500 student trainees, residents, and fellows. The combined School of Medicine and Medical System (“University of Maryland Medicine”) has an annual budget of over $6 billion and an economic impact of nearly $20 billion on the state and local community. The School of Medicine, which ranks as the 8th highest among public medical schools in research productivity (according to the Association of American Medical Colleges profile) is an innovator in translational medicine, with 606 active patents and 52 start-up companies. In the latest U.S. News & World Report ranking of the Best Medical Schools, published in 2021, the UM School of Medicine is ranked #9 among the 92 public medical schools in the U.S., and in the top 15 percent (#27) of all 192 public and private U.S. medical schools. The School of Medicine works locally, nationally, and globally, with research and treatment facilities in 36 countries around the world. Visit medschool.umaryland.edu. To learn more about Dr. Feldman’s research visit https://www.medschool.umaryland.edu/profiles/Feldman-Ricardo/

About The Michael J. Fox Foundation for Parkinson’s Research As the world’s largest nonprofit funder of Parkinson’s research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson’s disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson’s patients, business leaders, clinical trial participants, donors and volunteers. In addition to funding $1.5 billion in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson’s research, the Foundation forges groundbreaking collaborations with industry leaders, academic scientists and government research funders; increases the flow of participants into Parkinson’s disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson’s awareness through high-profile advocacy, events and outreach; and coordinates the grassroots involvement of thousands of Team Fox members around the world. For more information, visit us on at https://www.michaeljfox.org.

Forward-Looking Statements Any statements in this release that are not historical facts may be considered to be “forward-looking statements.” Forward-looking statements are based on management’s current expectations and are subject to risks and uncertainties which may cause results to differ materially and adversely from the statements contained herein. Such statements include, but are not limited to, statements regarding the market opportunity for Gain’s product candidates, and the business strategies and development plans of Gain. Some of the potential risks and uncertainties that could cause actual results to differ from those expected include Gain’s ability to: make commercially available its products and technologies in a timely manner or at all; enter into strategic alliances, including arrangements for the development and distribution of its products; obtain intellectual property protection for its assets; accurately estimate and manage its expenses and cash burn and raise additional funds when necessary. Undue reliance should not be placed on forward-looking statements, which speak only as of the date they are made. Except as required by law, Gain does not undertake any obligation to update any forward-looking statements to reflect new information, events or circumstances after the date they are made, or to reflect the occurrence of unanticipated events.

Investor & Media Contacts:

Gain Therapeutics Investor Contact: Daniel Ferry LifeSci Advisors +1 (617) 430-7576 daniel@lifesciadvisors.com

Gain Therapeutics Media Contact: Joleen Schultz Joleen Schultz & Associates +1 (760) 271-8150 joleen@joleenschultzassociates.com

Maryland Secretary of Commerce Kelly M. Schulz toured the new I-270 Innovation Labs on November 3rd, welcoming the much-needed center to the state’s tech and life sciences industries. The 22,000 square feet facility encompasses a wide mix of wet and dry labs, and is strategically located in southern Frederick County near both the I-270 and I-70 interstate highways.

“Having the private sector come forward and be able to create this kind of spec lab space in different parts of the state has been very, very helpful for the entrepreneurs,” Schulz said. “And we’ve seen a great growth in entrepreneurial resilience over the course of the pandemic in the last 20 months.”

Regional demand for sub-2,000 square feet laboratories has been chronically underserved, and the new I-270 Innovation Labs addresses this critical need with turnkey lab space that is highly adaptable and integrates essential operational safeguards. In particular, those needed to ensure high levels of biosafety. Forward thinking design brings about a range of high‐quality wet and dry laboratory layouts, and multimodal workspaces with rapid reconfiguration capabilities. On-demand key resources and services dovetail adaptable commercial terms so that enterprises, startups, research and educational institutions, and other types of tech and biotech organizations are able to reduce cost and risk, compress operational scaling and R&D timelines, and focus on discovery.

Click here to read more via BioBuzz.

Ellington West, CEO & Co-founder of Sonavi Labs, joins BioTalk to discuss their building the startup, their medical device, and raising funds in the BioHealth Capital Region.

Listen via Apple https://apple.co/3H25FPN, Google https://bit.ly/3kfXaXH, Spotify https://spoti.fi/3kgBLxI, and TuneIn https://bit.ly/3H43oU6.

Ellington West is an award-winning business leader, speaker, and advisor, recognized for being one of the very few Black American women to have raised more than $2 million for her startup venture. She is a 2021 Cartier Women’s Initiative North American Fellow, has been named to The Baltimore Sun’s 25 Women to Watch in Healthcare list, and is an advisor to the Smithsonian’s Lemelson Center for the Study of Invention and Innovation. Ellington dedicates her platform to advocating for diversity in executive roles, healthcare leadership, and venture funding, speaking most recently for the American Telehealth Association, Johnson & Johnson, and National Society of Black Engineers.

Click here for the transcript.

Early Charm’s continued portfolio company and staff growth drive need for more development and production space

November 2, 2021, Baltimore, MD – In a move to accommodate its continued fast growth, Early Charm – a company that creates, owns and operates ventures that convert science to revenue – announced today it is more than doubling its production and lab space at Baltimore’s 1100 Wicomico building.

This significant expansion will occur adjacent to Early Charm’s 3,200 square feet of current production and development space, bringing total production and development space to just under 7,000 square feet.

“Demand for our custom manufactured products is strong across the board with nanofibers, 3D printing, DNA libraries and custom inks experiencing the most rapid growth,” said Ken Malone, Executive Officer with Early Charm. “We’ve already removed the wall separating the spaces and will be able to use a large portion of the space immediately. The full build-out will only take a few months.”

Early Charm’s additional space will house an array of production and development equipment, including production-grade 3D printers, electrospinning machines to manufacture nanofibers, plus bench tops for life science development and analytical equipment.

Early Charm moved into 1100 Wicomico in February 2020 to accommodate its rapidly expanding custom manufacturing and development work for its portfolio of companies in Drug Design, Custom Materials, Blue Economy and Engineered Products.

About Early Charm:

Early Charm creates, owns and operates businesses that convert science into revenue. We are the management team, the operators and the entrepreneurs. We don’t write checks and just pop in for board meetings. We show up to our labs, our production facilities and our offices every day to do all the heavy lifting in our portfolio companies. Learn more: www.earlycharm.com

Media Contact:

Ken Berlack

Early Charm

ken.berlack@earlycharm.com

Amritha Jaishankar, PhD, Executive Director, Maryland Stem Cell Research Fund (MSCRF), joins BioTalk to discuss her career, investing in Maryland, and accelerating cures.

Listen via Apple https://apple.co/3ED5uIy, Google https://bit.ly/3EudFqG, Spotify https://spoti.fi/3EudHii, and TuneIn https://bit.ly/3bs9a3u.

Dr. Amritha Jaishankar serves as the Executive Director, Maryland Stem Cell Research Fund (MSCRF). In this role, Amritha is responsible for oversight of all MSCRF activities, programs, scientific management and oversight of the portfolio, as well as MSCRF business development. She works with stem cell scientists and a variety of key stakeholders to develop a collaborative research program and promote commercialization of stem cell technologies.

MSCRF has invested over $170 million in identifying, supporting, and accelerating cutting-edge research, innovation, clinical trials and commercialization of human stem cell-based technologies in Maryland. Amritha is scientist by training who has devoted her career to developing cures for debilitating and life-threatening conditions of our time and to advancing this field, through various roles in federal, university, and industry settings. She is passionate about using stem cell technologies to accelerate cures for patients in need. Her experience in and outside of the laboratory has made her a proven leader and champion for stem cell research and commercialization.

Since 2016, she has led an Accelerating Cures initiative at MSCRF and has helped create a vibrant and growing stem cell community in the region. She was recently recognized for her leadership in life sciences and contributions to the community by Women in Bio Capital Region and as a 2020 Leading Women honoree by The Daily Record, Maryland. Amritha serves on a number of advisory boards and committees in the region.

Download the transcript.