CRYAA

New Non-Clinical Research Highlights the Potential Role of Alpha-Crystallin Aggregation Inhibitors to Treat Cataract and Presbyopia SEATTLE & IRVINE, Calif.--(BUSINESS WIRE)-- Visus Therapeutics Inc., a clinical stage biopharmaceutical company focused on developing multi-targeted ophthalmic therapeutics for the front and back of the eye, today announced it will present new scientific data at the 2023 Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, to be held April 23-27, 2023 in New Orleans, Louisiana. The presentations will highlight new research findings on the Company’s alpha-crystallin aggregation inhibitor assets. Alpha-crystallin aggregation inhibitors are intended to restore elasticity and lens clarity, potentially reversing presbyopia and cataracts without the need for surgery. “We are pleased to share these scientific findings with the ophthalmic research community at ARVO 2023,” said Rhett Schiffman, M.D., M.S., M.H.S.A., co-founder, chief medical officer and head of research and development at Visus Therapeutics. “Our non-clinical work investigates the use of alpha crystallin chaperones as aggregation inhibitors for the potential treatment of cataract, the leading cause of global blindness, and presbyopia, which affects nearly every adult over the age of 50. Exploring non-surgical therapeutic options to treat these diseases has the potential to improve the quality of life for millions of people around the world.” The schedule of scientific presentations is as follows: Paper Title: Intravitreal α-Crystallin Chaperone Reverses Lens Opalescence in Non-Human Primates with Spontaneous Cataracts Date: Tuesday, April 25, 2023 Time: 9:15am – 9:30am (Central Time) Location: 343 Presenter: James Burke, Visus Therapeutics Poster Title: Next-Generation α-Crystallin Chaperones for the Treatment of Dysfunctional Lens Syndrome Date: Sunday, April 23, 2023 Time: 3:45pm – 5:30pm (Central Time) Location: Exhibit Hall Presenter: Patrick Hughes, Visus Therapeutics About Cataracts A cataract is a clouding of the natural lens of the eye, making it difficult to read, drive a car, or see faces. Cataracts become common with age; more than half of all Americans aged 80 or older have cataracts or have had surgery to remove them.1 About Visus Therapeutics With offices in Seattle, Wash., and Irvine, Calif., Visus Therapeutics is a clinical stage biopharmaceutical company focused on developing multi-targeted ophthalmic therapeutics for the front and back of the eye. The company is developing novel, pupil-modulating therapeutics designed to correct the loss of near vision associated with presbyopia. In parallel, Visus Therapeutics is advancing its pipeline of early-stage ophthalmic drug candidates engineered to preserve and restore visual function associated with the leading causes of vision loss including cataract and presbyopia, ocular surface and corneal disease, glaucoma, and age-related macular degeneration. For more information, visit and follow us on LinkedIn. _____________________ 1 Cataracts. Retrieved September 10, 2021. Johns Hopkins Medicine.

In January, BioSpace published our NextGen Bio Class of 2022 – a list of the top recently launched life sciences companies in North America. Now it’s time to check in with some of these companies, see what they’ve done to earn their place in the Class of ’22 and what’s on the horizon in 2023. Orna Therapeutics Located: Cambridge, MA Launched: February 2021 BioSpace Rank: 19 Series A: $80M Since Then: Orna, one of the most novel companies in BioSpace’s Class of ’22, had its circular RNA platform definitively validated in August when Merck poured a potential $3.5 billion into a collaboration deal. “Merck is an ideal partner given its scale and vaccine expertise,” said Orna CEO Tom Barnes, Ph.D. “Together, we'll explore the best application of our platform technology across a number of different pathogens in infectious disease and in oncology." As if that weren’t enough, Orna simultaneously announced a $221 million Series B financing round. Merck came on board as a new investor in the round. Merck had reason to believe: at the American Society of Gene and Cell Therapy's (ASGCT) 25th Annual Meeting, Orna announced first-ever data from its lead isCAR program validating both the technology and its LNP delivery platform. The in situ CAR therapy not only demonstrated tumor suppression but also eradication in a NALM6 (leukemia) mouse model. GentiBio Location: Boston, MA Launched: August 2021 BioSpace Rank: 6 Series A: $157M Since Then: After reeling in one of the largest Series A rounds of 2021, GentiBio has had another monster year. Named after the Latin word ‘Genti” which means “a congregation of peoples”, the company is congregating with peers at BMS in a nearly $2 billion deal to develop Treg therapies for inflammatory bowel disease. GentiBio is combining Tregs biology and antigen receptor engineering to develop engineered Tregs. Its hope is to provide a functional cure for autoimmune and autoinflammatory diseases. GentiBio has also been busy adding depth to its leadership team, with the appointments of Neely Mozaffarian, M.D., Ph.D. as chief medical officer and Chuck Silberstein, M.D. as chief financial officer. Enveda Biosciences Located: Boulder, CO Launched: June 2021 BioSpace Rank: 24 Series A: $51M Since Then: In April, Enveda announced a new mass spectrometry and automation laboratory in Boulder. The 60,000-square-foot facility is expected to be completed in the first quarter of 2023. Enveda simultaenously announced it would open a lead optimization lab in Genome Valley, Hyderabad, dubbed the “Life Science Capital of India.” Up Next: Enveda Founder and CEO Viswa Colluru told BioSpace the company expects to nominate differentiated development candidates in 2023 enabled by its access to unique chemistry. Enveda recently scaled its platform over 10X, “allowing us to find novel drug-like natural product leads at scale in any assay type,” Colluru said. With this scaling in place, the company “now [has] the bandwidth to pursue a partnership in addition to serving internal pipeline needs,” he added. To this end, Enveda has brought on Vanitha Sekar, Ph.D., R.Ph. as its first chief business officer. Eikon Therapeutics Located: Hayward, CA Launched: May 2021 BioSpace Rank: 3 Series A: $148M Since Then: When Eikon launched in May 2021, it did so with former Merck R&D Head Roger Perlmutter at the helm. In March, he was joined by Roy Baynes, Merck’s former SVP of global clinical development and CMO. Kenneth Frazier joined the board in April, making Eikon look a whole lot like Merck 2.0. The three have been guiding what Eikon calls its “groundbreaking approach to elucidating the behavior of proteins in live cells” ever since. Meanwhile, the company has been expanding its geographical footprint, pouring funds into a new corporate headquarters in San Francisco and a new operational base in New York City. Up Next: You? Eikon currently features 55 open jobs on BioSpace. Prime Medicine Location: Cambridge, MA Launched: July 2021 BioSpace Rank: 7 Series A: $115M Since Then: After launching with an impressive $315 million in cash, the prime editing company filed for an IPO in September. Prime expects to bring in $175 million, according to an October 19 announcemet. In July, Prime announced three appointments to its management team: Richard Brudnick as chief business officer, Niamh Alix as chief human resources officer and Fubao Wang, Ph.D., as SVP and head of regulatory. Prime has also been busy augmenting its platform. In March, the company inked an exclusive collaboration deal with Myeloid Therapeutics to accelerate the latter’s Myeloid's RetroT technology. Prime CEO Keith Gottesdiener, M.D. indicated the tech is complementary to Prime’s own gene editing toolbox. Umoja Biopharma Location: Seattle, WA Launched: November 2020 BioSpace Rank: 2 Series A: $53M Since Then: Umoja’s $263 million in Series A and B financing and quick expansion efforts were good for the No. 2 spot in BioSpace’s Class of ’22. Since then, the company has been busy refining its approach through partnerships with Lupagen, Inc. and TreeFrog Thearpeutics. Umoja has also presented preclinical data demonstrating the potential of its multi-pronged platform consisting of RACR/CAR, TumorTag and VivoVec. AavantiBio Location: Cambridge, MA Launched: October 2020 BioSpace Rank: 8 Series A: $107M Since Then: Sometimes a new company will go the distance, and other times a merger or acquisition seems like a better bet. AavantiBio found what appears to be a mutually beneficial path, announcing a merger with Solid Biosciences at the end of September. AavantiBio president and CEO Bo Cumbo will helm the new company, which will be know as Solid Biosciences and trade on the Nasdaq as SLDB. The company will focus its collective attention on progressing a portfolio of neuromuscular and cardiac programs, led by SGT-003, a differentiated gene transfer candidate for the treatment of Duchenne muscular dystrophy. Graphite Bio Location: South San Francisco, CA Launched: September 2020 BioSpace Rank: 12 Series A: $45M Since Then: In August, Graphite Bio dosed the first patient in the Phase I/II CEDAR trial investigating nulabeglogene autogedtemcel (nula-cel), an investigational gene correction therapy for sickle cell disease. The treatment was awarded FDA Fast Track Designation in May. In May, Graphite presented preclinical data from GPH102, its differentiated gene replacement program for beta-thalassemia, at ASGCT. The data showed GPH102 can restore the expression of these genes, providing a path forward for further development. Up Next? In mid-2023, Graphite anticipates sharing initial proof-of-concept data for nula-cel in sickle cell, CEO Josh Lehrer, M.D. revealed. The company also plans to share more information about its efforts to develop less harmful (non-genotoxic) conditioning regimens that are used as part of the genetic therapy treatment process. “We believe advances in this area will put less burden on the patients who elect to undergo treatment with genetic therapies and make these therapies available to greater numbers of patients in need,” Lehrer said. Visus Therapeutics Located: Seattle, WA Launched: March 2021 BioSpace Rank: 15 Series A: $36M Since Then: Visus has had a year worthy of its place on the list. The company’s ophthalmic pipeline grew in September when it acquired the patents and assets of ViewPoint Therapeutics. Visus picked up ViewPoint’s Alpha-crystallin aggregation inhibitor, a non-surgical option to treat protein misfolding in the eye. It has shown potential in primates to reverse age-related cataracts. It also has potential against presbyopia. In March, Visus launched two pivotal Phase III trials (BRIO-I and BRIO-II) for its lead asset, BRIMOCHOL PF, which is also in development for presbyopia. Metagenomi Located: Emeryville, CA Launched: November 2020 BioSpace Rank: 20 Series A: $65M Since Then: CRISPR is cool – just ask Leaps by Bayer and Humboldt Fund, the co-leaders of Metagenomi’s Series A. This year, Metagenomi has been busy on the conference circuit presenting preclinical data from its portfolio of next-generation gene editing tools at ASGCT and several other meetings. The company is moving quickly to ramp up the infrastructure for its programs. After closing a $175 million Series B round in January, Metagenomi recently opened a new, in-house GMP facility on its 50,000-square-foot campus in Emeryville. With Moderna as a partner, the future looks bright for Metagenomi.

Sept. 28, 2022 12:00 UTC Library of ViewPoint drug candidates will accelerate development of novel alpha-crystallin chaperone compounds as a non-surgical treatment for age-related cataracts. SEATTLE--(BUSINESS WIRE)--Visus Therapeutics Inc., (“Visus”) a clinical stage biopharmaceutical company focused on developing multi-targeted treatments for the eye, today announced it has expanded its pipeline via the acquisition of all patents and other assets of ViewPoint Therapeutics, Inc. (“ViewPoint”). Founded in 2014, ViewPoint discovered and developed alpha-crystallin aggregation inhibitors as investigational non-surgical treatments to correct protein misfolding in the lens. Protein misfolding reduces lens elasticity and clarity leading to presbyopia and cataract, the leading causes of visual disability worldwide. Presbyopia is an inability to see near objects clearly, due to a loss of lens elasticity, that begins in the 40s and inevitably worsens with age. As protein misfolding worsens, cataracts, or clouding of the lens, may develop that can affect all vision-related activities. Alpha-crystallin aggregation inhibitors are intended to restore elasticity and lens clarity, potentially reversing presbyopia and cataracts without the need for surgery. “We have already demonstrated that intraocular delivery of alpha-crystallin aggregation inhibitors reverses age-related cataracts in primates, giving us confidence that this approach may be effective in humans,” said Rhett M. Schiffman MD, MS, MHSA, the company’s co-founder, chief medical officer and head of research and development. “It may even be possible that intervening in presbyopia may not only restore near vision but prevent the development of cataracts in many patients. The ViewPoint acquisition provides an extensive library of potent drug candidates with highly desirable pharmaceutical properties for ocular delivery,” said Dr. Schiffman. Lead candidate selection is anticipated in Q1 2023. “Visus is currently conducting two Phase 3 pivotal studies of its preservative-free eyedrops, BRIMOCHOL™ PF and Carbachol PF, for the treatment of presbyopia. The ViewPoint assets are a natural complement to this late-stage program and will enable the company to further expand its pipeline targeting the leading causes of age-related loss of vision,” said Ben Bergo, co-founder and chief executive officer of Visus. “More than a third of the world’s population have presbyopia or cataracts, and this will only increase as the population ages. A non-surgical approach to treating and possibly preventing cataracts and presbyopia could improve the quality of life of millions of people, globally, and would be a disruptive addition to the management of our patients,” said Eric D, Donnenfeld, MD, Founding Partner, Ophthalmic Consultants of New York, Clinical Professor of Ophthalmology, New York University Medical Center, and Chair of Visus’ Medical Advisory Board. Visus looks forward to sharing additional information during the Eyecelerator meeting at the American Academy of Ophthalmology conference in Chicago, IL on Thursday, September 29th. About Presbyopia Presbyopia is the loss of near vision associated with aging, making it difficult to perform tasks like reading fine print. It typically begins when adults are in their 40s and becomes almost universal by age 50.i Presbyopia impacts billions of people globally with approximately 128 million adults affected in the U.S. alone.ii,iii Reading glasses are the most common solution for near-vision correction. However, many people find glasses inconvenient or prefer not to wear them for aesthetic reasons. About Cataracts A cataract is a clouding of the natural lens of the eye, making it difficult to read, drive a car, or see faces. Cataracts become common with age; more than half of all Americans aged 80 or older have cataracts or have had surgery to remove them.iv About Visus Therapeutics With offices in Seattle, Wash., and Irvine, Calif., Visus Therapeutics is a clinical stage biopharmaceutical company focused on developing multi-targeted treatments for the eye. The company is developing novel, pupil-modulating therapeutics designed to correct the loss of near vision associated with presbyopia. In parallel, Visus Therapeutics is focused on advancing its pipeline of early-stage ophthalmic drug candidates with applications in corneal wound healing, glaucoma and age-related macular degeneration. For more information, visit: and follow us on LinkedIn, Twitter, and Instagram. i U.S. Census Bureau. Retrieved September 7, 2019 from . ii Zebardast et al. The Prevalence and Demographic Associations of Presenting Near-Vision Impairment Among Adults Living in the United States. Am J Ophthalmol. 2017;174:134-144. iii U.S. Census Bureau. Table 9. Washington: Population Division. 2014. iv Cataracts. Retrieved September 10, 2021. Johns Hopkins Medicine.