Capsida Biotherapeutics, Inc.

LOS ANGELES, April 22, 2025 (GLOBE NEWSWIRE) -- Trethera Corporation (“Trethera”), a clinical stage biopharmaceutical company developing first-in-class therapies for cancer and autoimmune diseases, announces a forthcoming panel discussion and poster presentation at the 7th Annual Los Angeles Bioscience Ecosystem Summit 2025 (“LABEST”). Panel Discussion Details: Title: BioSuccess in Los AngelesCEO Panelists: Ken Schultz (Trethera), Daniel Gil (Pelage), Dina Leeds (CleopatraRx), and Bethany Mancilla (Capsida)Moderator: Amir Naiberg, Associate Vice Chancellor, UCLA; CEO and President, UCLA Technology Development GroupLocation: Centennial Ballroom, 1st floor, Luskin Conference CenterDate: Thursday, May 22, 2025 at 2PM Poster Details: Title: First-in-Class Clinical Stage Deoxycytidine Kinase Inhibitor Blocks Inflammatory Bowel Disease Symptoms in an Adoptive CD4 T Cell Transfer Mouse ModelAuthors: Peter M. Clark, PhD; Kenneth A. Schultz, MD; KM Ryan, BSCategory: Immunity, Inflammation, Infection and Transplantation (I3T)Location: Pearl Cohen Scientific Poster Competition, Luskin Conference CenterDate: Thursday, May 22, 2025 at 4PM LABEST 2025 is the premier showcase for bioscience innovation in Los Angeles County and is produced by the UCLA Technology Development Group. The conference attracts over 1,500 life science industry professionals from the world’s leading pharmaceutical companies, venture capital firms, and research institutions. LABEST is intended to promote awareness of the growing life science entrepreneurial ecosystem in Los Angeles and to foster partnerships with the biotechnology and life science industry. Poster co-author Peter M. Clark is an Associate Professor of Molecular and Medical Pharmacology at the David Geffen School of Medicine and Crump Institute for Molecular Imaging at UCLA. Dr. Clark identified the metabolic enzyme deoxycytidine kinase, dCK, as a key and targetable enzyme required for aberrant T and B cell activation in models of autoimmune diseases, such as Crohn’s disease and lupus. The Clark lab aims to develop novel, first-in-class, drug therapies for cancer and autoimmune diseases. Figure 1: Biochemical pathways for the supply of deoxyribonucleoside triphosphate pools. The salvage pathway becomes upregulated during autoimmune diseases. FDA approved drugs blocking the de novo pathway include Aubagio (teriflunomide), Mavenclad (cladribine), and CellCept (mycophenolate mofetil). About TretheraTrethera is a clinical stage, privately held, biopharmaceutical company dedicated to pioneering the development of novel treatments for autoimmune diseases and cancers. Founded by prominent UCLA scientists, Trethera is led by experienced management and board members. Trethera's innovative approach to targeting nucleotide metabolism led to the development of TRE-515, an orally administered capsule twice designated by the FDA as an Orphan Drug. TRE-515 is a first-in-class clinical stage drug that inhibits deoxycytidine kinase (dCK), the rate-limiting enzyme in the nucleoside salvage pathway, one of two biosynthetic pathways that generate DNA precursors. It is believed that some forms of cancer may be preferentially dependent on the salvage pathway to support tumor growth, and certain autoimmune diseases might also respond to TRE-515 treatment. Trethera is developing TRE-515 for use as a monotherapy or in combination to precisely target a metabolic vulnerability of cancer or autoimmune diseases that will transform outcomes for patients. For more information, please visit us at trethera.com or e-mail Investor Relations at ir@trethera.com. You can also follow Trethera on Facebook and LinkedIn. Note on Forward-Looking StatementsAll statements other than statements of historical facts included in this press release that address activities, events or developments that Trethera believes or anticipates will or may occur in the future are “forward-looking statements,” which may often, but not always, be identified by the use of such words as "may," "might," "will," "will likely result," "would," "should," "estimate," "plan," "project," "forecast," "intend," "expect," "anticipate," "believe," "seek," "continue," "target" or the negative of such terms or other similar expressions. Although Trethera has a reasonable basis for the forward-looking statements contained herein, Trethera cautions that such statements are based on current expectations about future events and are subject to risks, uncertainties and factors relating to medical and scientific research, all of which are difficult to predict and many of which are beyond Trethera’s control, that may cause actual results to differ materially from those expressed or implied by the forward-looking statements in this press release. These potential risks and uncertainties include, without limitation: the extent to which development of any novel cancer therapies or therapies for autoimmune diseases succeeds; whether Trethera would obtain the necessary regulatory approvals to commence human trials or commercialize TRE-515 or any novel therapies resulting from such research; Trethera successfully implementing its growth strategy, including that relating to its disease therapies; the effects of the global Covid-19 pandemic; changes in economic conditions; competition; and risks and uncertainties applicable to the business of Trethera. The statements in this press release speak only as of the date hereof and Trethera does not undertake any obligation to update, amend or clarify these forward-looking statements whether as a result of new information, future events or otherwise. The Company intends that all forward-looking statements be subject to the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995. A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/a3669834-fb66-4439-930d-931d3a67f1b7

Sangamo has made another gene therapy deal to keep the lights on for a few more months, extending its funding by another three months until at least mid-2025. On Thursday, the struggling California biotech said it would license its experimental brain-targeting adeno-associated virus capsid to Astellas for $20 million in cash, plus milestone payments, for an undisclosed neurological disease target. The virus is designed to cross the blood-brain barrier that keeps most drugs out. It’s the second time this year Sangamo has struck an AAV deal that adds to its slim bank account and delays a possible bankruptcy. In March, it told investors it only had enough funds to survive into the third quarter. In August, a lifeline came through a partnership with Genentech , which paid $50 million upfront for preclinical-stage gene therapies for Alzheimer’s and an undisclosed condition, both reliant on Sangamo’s new AAVs. Although existing capsids like AAV9 can be injected directly into the spinal cord or brain, developing delivery vehicles that can cross into the brain from a simple blood infusion has proven difficult. Sangamo has said its new capsid, STAC-BBB, was 700 times better at expressing genes in the brains of monkeys compared to AAV9. Several biotech companies, including Affinia Therapeutics, Capsida Biotherapeutics, Dyno Therapeutics, Voyager Therapeutics and others, are racing to develop brain-penetrant capsids. An Astellas spokesperson said that after an “an extensive evaluation,” the company decided Sangamo’s was “a best-in-class capsid for the treatment of serious genetic diseases” of the central nervous system. The Genentech money was enough to sustain Sangamo until the first quarter of 2025. A Sangamo spokesperson told Endpoints News that with the Astellas license, “combined with other potential levers in development, Sangamo has line of sight to extending the cash runway into Q2 2025 and beyond.” Astellas could expand its work on up to four more targets, and Sangamo could earn up to $1.3 billion in license fees and milestone payments across the five potential programs. On Wednesday evening, Sangamo’s stock $SGMO was up roughly 370% since the start of 2024, buoyed by its October announcement that it plans to ask for an accelerated approval of its gene therapy for Fabry disease, without the need for a confirmatory study, next year. Its stock also jumped this week when Roche announced it would halt development of a competing gene therapy for hemophilia A. But the company’s $550 million market cap is still among the lowest in its 24 years as a public company and is down 82% since its IPO in 2000.

The therapy restored syntaxin-binding protein 1 (STXBP1) protein levels in primate neurons and in lab-cultured human neurons. STXBP1 is one of the genes that can cause DEE when mutated.\n Capsida Biotherapeutics has unveiled data showing that its AAV-delivered gene therapy for developmental and epileptic encephalopathy (DEE), the most severe form of epilepsy, corrected cognitive and motor deficits and reduced seizures in mice up to one year after treatment.The California-based biotech presented the results on Dec. 7 at the annual meeting of the American Epilepsy Society in Los Angeles.Capsida recently met with the FDA for a pre-Investigational New Drug meeting and received an Orphan Drug Designation for the therapy, called CAP-002, the company said in a Dec. 6 release. Capsida plans to bring CAP-002 into the clinic in the first half of 2025. The therapy restored syntaxin-binding protein 1 (STXBP1) protein levels in 70% of primate neurons, according to the abstract. STXBP1 is one of the genes that can cause DEE when mutated. CAP-002 also restored STXBP1 protein levels and neuronal network activity in human neurons cultured in the lab, Capsida said.“STXBP1-DEE is a life-altering condition for patients and their families, and there are no approved treatments,” Capsida CEO Peter Anastasiou said in the release. “These new data demonstrate the potential of CAP-002 to safely address all of the manifestations of the disease, and we look forward to progressing this program into the clinic in the first half of 2025.”DEE often manifests in infancy, with children experiencing frequent seizures that are difficult to control with typical anti-seizure medications. Patients commonly have other conditions alongside DEE, like sleep problems, movement disorders and autism spectrum disorder.