Suzetrigine

USA News Group News Commentary Issued on behalf of Avaí Bio, Inc. As the broader cell and gene therapy complex posts record quarterly revenue and advances toward accelerated approval pathways, a Nevada-based longevity joint venture has just cleared a manufacturing-stage gate on its α-Klotho anti-aging program. NEW YORK, May 28, 2026 /PRNewswire/ -- Cell and gene therapy has crossed a meaningful inflection in 2026. Vertex Pharmaceuticals reported Q1 product revenue of $2.99 billion, with newer non-CF franchises CASGEVY and JOURNAVX contributing more than 25% of year-over-year growth. Alnylam Pharmaceuticals delivered its first-ever $1 billion product revenue quarter. Beam Therapeutics presented updated BEAM-302 clinical data at ATS 2026 and announced a pivotal expansion under an FDA-aligned accelerated approval pathway. Sana Biotechnology entered a strategic Mayo Clinic collaboration including a $25 million equity investment. Inside that landscape, Avaí Bio, Inc. (OTCQB: AVAI), Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX), Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY), Beam Therapeutics Inc. (NASDAQ: BEAM), and Sana Biotechnology, Inc. (NASDAQ: SANA) collectively represent the breadth of where cell-based and engineered-cell platforms are converting science into clinical and commercial milestones. Avaí Bio, Inc. (OTCQB: AVAI) just delivered one of the most important production-stage milestones in its corporate history. On May 20, 2026, Avaí Bio and joint venture partner Austrianova announced the completion of a GMP-grade Master Cell Bank (MCB) of genetically modified cells overexpressing the α-Klotho protein. The MCB will now undergo comprehensive third-party testing to confirm the absence of pathogenic viruses and adventitious agents — bacteria, mycoplasma, fungi, and yeast — to meet regulatory expectations including FDA standards. The completion of the GMP MCB is a prerequisite gate for Klothonova's path toward preclinical and clinical advancement of α-Klotho cell therapy. Klothonova, the joint venture established in September 2025 and operationalized as a Nevada-based corporation equally owned by Avaí Bio and Austrianova's affiliate SG Austria Pte. Ltd., is dedicated to the sustainable production of α-Klotho — widely recognized in the scientific literature as the "longevity protein" — using encapsulated cell-based therapies. The therapeutic approach is designed to address general aging biology as well as age-related conditions including kidney disease, neurodegenerative disorders, and vascular diseases. The combination of Avaí Bio's biotechnology focus and Austrianova's Cell-in-a-Box® cell-encapsulation intellectual property and GMP manufacturing capability creates an integrated development-to-clinic structure rare among small-cap longevity-focused biotech. Chris Winter, Chief Executive Officer of Avaí Bio, framed the program direction in the Company's release, noting completion of the first production phase as a step toward delivering safe, effective treatments for aging-associated diseases. Brian Salmons, CEO of Austrianova, added that the Austrianova team is pleased to have completed production of the α-Klotho-expressing cells on behalf of Klothonova, with subsequent steps directed at producing the encapsulated Cell-in-a-Box® clinical product for patient application. The data narrative is reinforced by additional upcoming visibility. On April 7, 2026, Avaí Bio and Austrianova announced that the latest data from the α-Klotho anti-aging therapy program will be presented at the Second Annual Klotho Conference in September 2026, with Rahul Pawa, Chief Production Officer at Austrianova, scheduled to deliver the presentation representing Avaí Bio, Austrianova, and Klothonova. The α-Klotho program — focused on sustainably restoring circulating α-Klotho levels via encapsulated cells — is positioned alongside Avaí Bio's complementary Insulinova diabetes program as the cornerstones of the Company's emerging cell-based therapeutic platform. Avaí Bio is an emerging biotechnology company developing transformative cell-based therapies for diabetes, age-related disorders, and anti-aging. The Company's commercial structure pairs in-house program ownership with manufacturing access via the Austrianova partnership and the Klothonova joint venture. With the GMP MCB now complete, viral testing initiated, and a September conference presentation scheduled, Avaí Bio is moving from preparatory work into the documented testing and preclinical advancement phase of its lead anti-aging program. For more information, visit the AVAI profile page. In other industry developments and happenings in the market include: Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) reported Q1 2026 financial results on May 4, 2026, with total revenues of $2.99 billion, up 8% year-over-year. The Company's cystic fibrosis franchise generated $2.92 billion, with TRIKAFTA/KAFTRIO at $2.35 billion and newer entrant ALYFTREK contributing $424 million — bringing cumulative ALYFTREK sales above $1 billion since its December 2024 U.S. launch. CRISPR-edited cell therapy CASGEVY delivered $43 million in revenue and crossed more than 500 patients initiated, while novel non-opioid acute pain medicine JOURNAVX generated $29 million on more than 350,000 scripts. CEO and President Reshma Kewalramani, M.D. framed the quarter as evidence of an increasingly diversified revenue base, with CASGEVY and JOURNAVX contributing more than 25% of revenue growth. Vertex reiterated full-year 2026 revenue guidance of $12.95–$13.1 billion, including at least $500 million from non-CF products. Net income rose to $1.03 billion (from $646.3 million in Q1 2025), and the Company closed the quarter with approximately $13.0 billion in cash — among the strongest balance sheets in cell therapy commercialization. Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) reported its Q1 2026 financial results on April 30, 2026, with net product revenues of $1.036 billion — up 121% year-over-year and the first $1 billion product revenue quarter in the Company's history. Total revenues reached $1.17 billion (+96% YoY), with GAAP net income of $206 million. AMVUTTRA, Alnylam's lead RNAi therapy for transthyretin amyloid cardiomyopathy (ATTR-CM), generated $889.9 million in Q1, supporting full-year 2026 TTR franchise guidance of $4.4–$4.7 billion. CEO Yvonne Greenstreet, M.D. described the quarter as positioning Alnylam for sustained long-term growth as the leader in RNAi therapeutics. The Company also initiated a Phase 1 trial of ALN-2232, its first adipose-directed RNAi therapeutic, targeting ACVR1C as a candidate for obesity and weight management — a strategic pipeline expansion into a high-value therapeutic area. Alnylam reiterated full-year 2026 total net product revenue guidance of $4.9–$5.3 billion and is advancing more than 25 clinical programs across rare disease, cardiometabolic, and CNS indications. Beam Therapeutics Inc. (NASDAQ: BEAM) on May 18, 2026 presented updated clinical data from its Phase 1/2 trial of BEAM-302 — a liver-targeting lipid-nanoparticle base-editing therapy for alpha-1 antitrypsin deficiency (AATD) — at the American Thoracic Society International Conference. Treatment with BEAM-302 led to rapid and durable increases in total and functional AAT, decreases in mutant Z-AAT, and new production of corrected M-AAT, with a well-tolerated safety profile across single doses up to 75 mg. Based on FDA feedback, Beam is pursuing an accelerated approval pathway for BEAM-302 based on a primary endpoint of AAT biomarkers evaluated over 12 months at the 60 mg selected dose. To support a future biologics licensing application, Beam anticipates enrolling approximately 50 additional patients with AATD-associated lung disease in an expansion of the ongoing open-label trial, with the pivotal cohort expected to initiate in the second half of 2026. CEO John Evans, who was recognized on the TIME100 Health list earlier in 2026, and Beam Therapeutics — selected for the TIME100 Most Influential Companies list — are advancing one of the more visible gene-editing-enabled accelerated regulatory pathways in the sector. The BEAM-302 expansion represents the type of regulatory derisking that the broader cell and gene therapy complex has needed at the clinical-to-commercial transition. Sana Biotechnology, Inc. (NASDAQ: SANA) reported Q1 2026 results in early May, highlighting a strategic collaboration with Mayo Clinic to advance development of SC451, a hypoimmune (HIP)-modified, induced pluripotent stem cell (iPSC)-derived pancreatic islet cell therapy for type 1 diabetes. In connection with the collaboration, Mayo Clinic made an approximately $25.0 million equity investment in Sana, reflecting a shared commitment to advancing innovative approaches for type 1 diabetes care. The Company also continues to update its lead UP421 program — an allogeneic primary islet cell therapy engineered with HIP technology, transplanted into a type 1 diabetes patient at Uppsala University Hospital without any immunosuppression. As of the most recent 14-month follow-up, UP421 has continued producing insulin (measured by circulating C-peptide levels) and maintained pancreatic islet cell function. President and CEO Steve Harr, M.D. has framed the data as reinforcing Sana's capability to develop a functional cure for type 1 diabetes — and as a clinical proof-of-concept for hypoimmune engineering across allogeneic cell therapies. Across the comparable set, the message is consistent: cell and gene therapy has moved from frontier science to commercial reality, with billion-dollar quarters at the senior end of the sector and accelerated regulatory pathways unlocking value at the clinical-stage end. Avaí Bio's May 20 GMP Master Cell Bank completion places the Klothonova α-Klotho program inside that broader commercial logic — with viral testing now underway, conference visibility planned for September, and a Cell-in-a-Box® manufacturing pathway anchored by partner Austrianova. For investors building exposure to the cell-based longevity therapeutics opportunity, AVAI deserves a closer look. CONTINUED… Read this and more news for Avaí Bio at: CONTACT: USA News Group [email protected] 604-265-2873 Article Sources: [1] [2] [3] [4] [5] [6] DISCLAIMER: Nothing in this publication should be considered as personalized financial advice. We are not licensed under securities laws to address your particular financial situation. No communication by our employees to you should be deemed as personalized financial advice. Please consult a licensed financial advisor before making any investment decision. This is a paid advertisement and is neither an offer nor recommendation to buy or sell any security. We hold no investment licenses and are thus neither licensed nor qualified to provide investment advice. The content in this report or email is not provided to any individual with a view toward their individual circumstances. 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– Suzetrigine, a selective pain signal inhibitor, has the potential to treat millions of Canadians who suffer from moderate-to-severe acute pain each year – TORONTO , May 21, 2026 /CNW/ - Vertex Pharmaceuticals (Nasdaq: VRTX) today announced that Health Canada has accepted for review a New Drug Submission (NDS) for suzetrigine for the treatment of moderate-to-severe acute pain in adults. Suzetrigine is a selective NaV1.8 pain signal inhibitor that represents a new class of oral pain medicine; it is not an opioid or a nonsteroidal anti-inflammatory drug (NSAID). If approved by Health Canada, suzetrigine has the potential to be the first new class of medicine in Canada to treat acute pain in over twenty years. Each year, approximately 8 million Canadian adults are prescribed pain medication to manage their acute pain. "Today's acceptance of our NDS for suzetrigine represents an important step in our mission to transform pain management in Canada," said Michael Siauw, General Manager at Vertex Pharmaceuticals (Canada) Incorporated. "Canadians experiencing acute pain deserve access to innovative treatment options. After more than two decades without a new class of acute pain medicine, suzetrigine has the potential to address this significant unmet need and expand the options available to health care providers." Vertex intends for the regulatory review of suzetrigine to be part of an aligned review with Canadian Health Technology Assessment (HTA) organizations: Canada's Drug Agency (CDA-AMC) and the Institut national d'excellence en santé et en services sociaux (INESSS) in Quebec. Aligned reviews provide an opportunity for information sharing between Health Canada and HTA bodies, leading to greater coordination to support Canadians' timely access to effective new therapies. Suzetrigine was approved by the U.S. Food and Drug Administration under the trade name JOURNAVX ® in January 2025. The U.S. application received breakthrough therapy, fast track, and priority review designations. About Suzetrigine Suzetrigine (JOURNAVX) is an investigational, oral, selective NaV1.8 pain signal inhibitor. NaV1.8 is a voltage-gated sodium channel that is selectively expressed in peripheral pain-sensing neurons (nociceptors), where its role is to transmit pain signals (action potentials). NaV1.8 is not expressed in the human brain and is not related to addiction. NaV1.8 is a genetically validated target for the treatment of pain, and suzetrigine has demonstrated a favorable benefit/risk pro three Phase 3 studies and two Phase 2 studies in patients with moderate-to-severe acute pain. The U.S. Food and Drug Administration approved twice-daily JOURNAVX for the treatment of adults with moderate-to-severe acute pain, including postoperative pain. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including IgA nephropathy, neuropathic pain, APOL1-mediated kidney disease, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes, generalized myasthenia gravis, and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 16 consecutive years on Science magazine's Top Employers list and one of Fortune's 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit . Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Michael Siauw in this press release, statements regarding the expected benefits of suzetrigine, including its potential to be the first new class of medicine in Canada to treat acute pain in over twenty years, and statements regarding expectations for the aligned review with the HTA, CDA-AMC and the INESSS. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that regulatory authorities in Canada may not approve suzetrigine on a timely basis or at all, and other risks listed under the heading "Risk Factors" in Vertex's annual report and in subsequent filings filed with the Securities and Exchange Commission and available through the company's website at and . You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. (VRTX-GEN) Vertex Pharmaceuticals Incorporated Investors: InvestorInfo@vrtx.com Media: mediainfo@vrtx.com or Canada: +1 647-790-1600 SOURCE Vertex Pharmaceuticals (Canada) Inc.