Hereditary retinal dystrophy

DURHAM, N.C., Jan. 16, 2024 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to ATSN-101, the company’s investigational gene therapy being evaluated in an ongoing Phase I/II clinical trial in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1). The FDA previously granted Regenerative Medicine Advanced Therapy (RMAT) designation and orphan drug designation to ATSN-101 for the treatment of LCA1. RPD designation is granted by the FDA for serious or life-threatening diseases which affect fewer than 200,000 people in the United States and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age. If a Biologics License Application for ATSN-101 for the treatment of LCA1 is approved by the FDA, Atsena may be eligible to receive a Priority Review Voucher that can be redeemed to receive a priority review for any subsequent marketing application or may be sold or transferred. “Rare Pediatric Disease designation is a significant milestone for our LCA1 program as we explore options to advance ATSN-101 into a pivotal clinical trial,” said Patrick Ritschel, MBA, Chief Executive Officer of Atsena Therapeutics. “The FDA designations that have been granted to ATSN-101 not only emphasize the tremendous need for a treatment for patients with LCA1, but also the potential for our subretinal gene therapy to be a major advance in reversing pediatric blindness.” Positive 12-month safety and efficacy data from the company’s ongoing Phase I/II clinical trial of ATSN-101 will be presented at the 47th Annual Macula Society Meeting, which is being held February 7-10, 2024, in Palm Springs, CA. ATSN-101 has demonstrated clinically meaningful improvements in vision at the highest dose and is well-tolerated 12 months post-treatment. Details of the presentation are as follows: Title: Twelve-month safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1) Session: Inherited Retinal Dystrophy I (Treatment Trials) Date and Time: Wednesday, February 7, 2024, 6:04 p.m. PST Presenter: Christine Nichols Kay, MD About GUCY2D-associated Leber congenital amaurosis (LCA1) LCA1 is a monogenic eye disease that disrupts the function of the retina. It is caused by mutations in the GUCY2D gene and results in early and severe vision impairment or blindness. GUCY2D-LCA1 is one of the most common forms of LCA, affecting roughly 20 percent of patients who live with this group of inherited retinal diseases. There are currently no approved treatments for LCA1. About Atsena Therapeutics Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company has two clinical-stage programs, ATSN-201 for X-linked retinoschisis (XLRS) and ATSN-101 for GUCY2D-associated Leber congenital amaurosis (LCA1). ATSN-201, which leverages the company’s novel spreading capsid AAV.SPR, is being evaluated in XLRS patients in a Phase I/II clinical trial known as the LIGHTHOUSE study. The company’s additional proprietary asset is ATSN-301, a dual AAV vector-based gene therapy to prevent blindness from MYO7A-associated Usher syndrome (USH1B). Interim safety and efficacy data from the company’s ongoing Phase I/II clinical trial in patients with LCA1 have demonstrated ATSN-101 is well tolerated and clinically meaningful improvements in vision were observed 12 months post-treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise. For more information, please visit atsenatx.com. Media Contact: Tony Plohoros 6 Degrees (908) 591-2839 tplohoros@6degreespr.com Business Contact: info@atsenatx.com

CHINO HILLS, Calif., Jan. 09, 2024 (GLOBE NEWSWIRE) -- via IBN -- SOHM, Inc. (OTC PINK: SHMN) (the “Company”), a pharmaceutical, nutraceutical, cosmeceutical and biotech Company that manufactures and markets generic drugs covering numerous treatment categories, announces today the Company has filed a provisional patent application on its ABBIE “Smart Gene-Editing” platform that uses the latest enzymes and guide RNAs to resolve off-target gene-editing limitations. Gene-editing Market The gene-editing market was valued at $5.3 billion in 2023. The gene-editing market is expected to grow to $10.8 billion by 2028 with a 15% CAGR. (Source: 2023, Marketsandmarkets.com.) “The U.S. FDA defines gene therapy as a treatment ‘to modify or manipulate the expression of a gene or to alter the biological properties of living cells for therapeutic use.’ Gene therapy has become a critical tool for treating genetic diseases …,” according to Life Sciences Intelligence. The top five most expensive gene-editing therapies approved by the FDA are: -- Hemgenix for hemophelia B ($3.5M / tx, approved Nov 2022) -- Skysona for active cerebral adrenoleukodystrophy ($3M / tx, approved Sept 2022) -- Zynteglo for beta-thalassemia ($2.8M / tx, approved Aug 2022) -- Zolgensma for spinal muscular atrophy ($2.1M / tx, approved May 2019) -- Luxturna for hereditary retinal dystrophy ($850,000 / eye, approved Dec 2017) (Source: Life Sciences Intelligence, May 2023) Drawbacks of Off-Target Edits All effective gene therapies require a high level of control so that the gene-edits happen at precisely the intended site, and not “off-target” at unintended sites. At any stage of the genomic edit, off-target edits in clinical settings can bear considerable consequences such as: -- Tissue damage -- Transformation of healthy cells into cancerous cells The dangers above are amplified by the difficulty of detecting off-target edits which can lead to significant consequences for the administered gene-therapies, like those listed above. The dangers and risks of off-target gene-edits are universal among leading gene-editing technologies including the likes of CRISPR. All gene-editing technologies currently struggle with their lack of control and precision clinical applications (1). ABBIE Resolves Off-target Edits and Improves Precision ABBIE is resolving off-target edits by using the latest advances in: -- Enzymes, -- Guide RNA and -- Cell or tissue-specific delivery. ABBIE is able to significantly improve gene-editing precision with improved systems for DNA insertion. ABBIE’s cell engineering tools and systems can reduce incidences of off-target edits by: -- not inducing double-stranded breaks (2) and -- provide the power of retroviral integration without using a virus. ABBIE Provisional Patent Filings SOHM’s developers have recently submitted a provisional patent application detailing technology designed to more tightly control editing events. This patent pending ABBIE technology will leverage effector molecules, such as kinases and phosphatases, to regulate the activity of engineered DNA at a target site. ABBIE will also utilize advances in graphene-based nanomaterials, nano sensors, and wireless communication technologies allowing for enhanced control and fine-tuning of the gene editing process. Graphene-based nanomaterials (3) will revolutionize biotechnology, diagnostics and medicine. The use of graphene nanoparticles in genome editing offers several key advantages: 1. Enhanced Delivery Efficiency: Graphene nanoparticles can efficiently deliver gene-editing tools into target cells, ensuring a higher success rate in modifying the genome. Their small size and surface functionalization enable effective cellular uptake and intracellular delivery. 2. Improved Precision: The high surface area and stability of graphene nanoparticles allow for precise binding and interaction with target DNA sequences. This ensures that the gene-editing tools are delivered to the intended genomic sites, minimizing off-target effects and increasing the accuracy of modifications. 3. Reduced Immune Response Graphene nanoparticles have shown excellent biocompatibility, reducing the risk of triggering an immune response. This is crucial for successful genome editing, as immune reactions can hinder the efficiency and safety of the process. 4. Versatile Applications The use of graphene nanoparticles in genome editing is not limited to human health. It has the potential to revolutionize agriculture, environmental remediation, and other fields where precise genetic modifications are desired. SOHM is at the forefront of this groundbreaking technology, working closely with leading researchers and institutions to advance the field of genome editing using graphene nanoparticles. The company's team of experts is committed to developing safe and effective solutions that will transform the way genetic diseases are treated and pave the way for exciting new discoveries in the field of biotechnology. "We are thrilled to introduce this game-changing technology to the world," said Dr. David Aguilar, Ph.D., SOHM’s COO. "Graphene nanoparticles have the potential to revolutionize genome editing, offering unprecedented precision and efficiency. We believe this breakthrough will have a profound impact on human health and open up new possibilities for scientific research as more gene therapies continue to pass FDA approvals." SOHM has filed a provisional patent application on its innovative technology. REFERENCES (1) Keep off-target effects in focus | Nature Medicine(2) Modulation of DNA double-strand break repair as a strategy to improve precise genome editing | Oncogene (nature.com)(3) Ioannis Prattis, Ernestine Hui, Patrik Gubeljak, Gabriele S. Kaminski Schierle, Antonio Lombardo, Luigi G. Occhipinti, Graphene for Biosensing Applications in Point-of-Care Testing, Trends in Biotechnology, Volume 39, Issue 10, 2021, Pages 1065-1077. About SOHM, Inc. SOHM, Inc. is a growing generic pharmaceutical manufacturing and marketing company with a vision of “Globalè Prospèro” that aims to build continuous growth. SOHM manufacturing and marketing targets rapidly growing healthcare segments such as nutraceuticals, cosmeceuticals and other major therapeutic segments. SOHM is headquartered in North America with manufacturing alliances in India and strategic alliances with U.S. manufacturing facilities. Although SOHM’s generic pharmaceuticals are exported globally and were introduced to the USA in early 2013, SOHM continues its focus on distribution to emerging markets in Africa, Latin America and Southeast Asia. To learn more about SOHM, Inc., visit www.SOHM.com. Safe Harbor Statement: This news release contains “forward-looking statements,” which are statements that are not purely historical and include any statements regarding beliefs, plans, expectations or intentions regarding the future. Such forward-looking statements include, among other things, the development, costs and results of new business opportunities. Actual results could differ from those projected in any forward-looking statements due to numerous factors. Such factors include, among others, the inherent uncertainties associated with new projects and development-stage companies. These forward-looking statements are made as of the date of this news release, and we assume no obligation to update the forward-looking statements or to update the reasons why actual results could differ from those projected in the forward-looking statements. Although we believe that any beliefs, plans, expectations and intentions contained in this press release are reasonable, there can be no assurance that any such beliefs, plans, expectations or intentions will prove to be accurate. Investors should consult all of the information set forth herein and should also refer to the risk factors disclosure outlined in our annual report for the most recent fiscal year and our quarterly reports uploaded from time to time on OTCMarkets.com. For more information, please contact:SOHM, Inc.+1 (714) 522-6700 Corporate Communications:InvestorBrandNetwork (IBN)Los Angeles, Californiawww.InvestorBrandNetwork.com+1 310.299.1717 OfficeEditor@InvestorBrandNetwork.com

SEONGNAM-SI, South Korea , Nov. 10, 2023 /PRNewswire/ -- Rznomics Inc., a South Korea based biopharmaceutical company specialized in the development of RNA-based gene therapeutics, announced today that it has received Fast Track Designation from the U.S. Food and Drug Administration (FDA) for RZ-001, for the treatment of patients with Glioblastoma (GBM). Fast Track Designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and address unmet medical needs, thus enabling drugs to reach patients sooner. If relevant criteria are met, the drug with Fast Track Designation may also be eligible for Accelerated Approval and Priority Review. GBM is known as the most malignant tumor in Central Nervous system with high mortality rate but lacks effective therapies. RZ-001, the RNA replacement enzyme-based cancer gene therapy for the treatment of GBM, targets and cleaves hTERT mRNA and replaces the mRNA with the therapeutic gene RNA. This induces anti-cancer activity and cytotoxic effect by trans-ligating an HSVtk-encoding sequence into the reprogrammed hTERT mRNA. RZ-001 has demonstrated very promising responses in preclinical studies in which an enhanced anti-cancer efficacy and a higher survival rate was observed. "We are proud to receive Fast Track Designation from the FDA," said Dr. Seong-Wook Lee, Chief Executive Officer of Rznomics. "This designation signifies an important milestone in developing a safe and effective treatment for patients with GBM who are in need of new therapeutic options." Rznomics received Phase I/IIa IND approval for RZ-001 from the FDA and the South Korean Ministry of Food and Drug Safety (MFDS) in Glioblastoma and the clinical trial will investigate the safety, tolerability, and efficacy of RZ-001 in patients with GBM. Subjects will be treated with RZ-001 according to the planned dose escalation design. About RZNOMICS As a biopharmaceutical company founded in the laboratory of Professor Seong-Wook Lee, Dankook University Department of Bioconvergence Engineering, Rznomics is researching with the goal of developing new RNA-based gene therapeutic bio-drugs for cancer and incurable diseases. Rznomics' core platform technology is based on an RNA replacement enzyme, known as trans-splicing ribozyme, which can edit target RNA through simultaneous destruction and repair (and/or reprogramming) to yield the desired therapeutic RNA, thus, selectively inducing therapeutic gene activity in cells expressing the target RNA. Rznomics' lead candidate (RZ-001) is a treatment for Hepatocellular Carcinoma & Glioblastoma (Phase 1/2a IND approval from both in South Korea and in the U.S.), with other treatments for Alzheimer's disease and hereditary retinal dystrophy (RD) For more information, please visit SOURCE Rznomics Inc.