Gene Therapy: 5 Biotech Stocks Chasing the $36B Prize

Canada NewsWire

VANCOUVER, BC, Jan. 2, 2026

Issued on behalf of Avant Technologies Inc.

VANCOUVER, BC, Jan. 2, 2026 /CNW/ -- Equity Insider News Commentary – The gene therapy market is surging toward $36.55 billion by 2032, powered by rising demand for curative treatments that target genetic diseases at their source^[1]. The FDA approved three transformative cell therapies this December alone, marking an inflection point where living medicine moves from experimental concept to commercial reality^[2]. This revolution in restorative biology defines the pipelines of Avant Technologies, Inc. (OTCQB: AVAI), Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX), CRISPR Therapeutics (NASDAQ: CRSP), Prime Medicine, Inc. (NASDAQ: PRME), and Madrigal Pharmaceuticals, Inc. (NASDAQ: MDGL).

The cell and gene therapy sector is projected to reach $39.61 billion by 2034, growing at a 17.98% compound annual rate as precision-based therapies address previously untreatable genetic disorders^[3]. Manufacturing breakthroughs in viral vector delivery are now enabling scalable production of these therapies, driving capital inflows from investors betting on functional cures rather than lifelong symptom management^[4].

Avant Technologies, Inc. (OTCQB: AVAI) is developing cell-based therapies for diabetes, aging, and chronic diseases using a proprietary encapsulation technology that protects genetically modified therapeutic cells from immune rejection. The company operates through two strategic joint ventures focused on addressing massive global health markets.

The first, Insulinova, Inc., is a partnership with SGAustria Pte. Ltd., through which Avant is developing treatments for type 1 and insulin-dependent type 2 diabetes. The approach centers on a cell encapsulation technology that allows genetically modified insulin-producing cells to survive long-term in the body without being susceptible to any immune system attacks. This solves a fundamental problem that historically required patients to take lifelong immunosuppressive drugs with serious side effects.

"Cell encapsulation is a game-changer in the field of regenerative medicine," said Chris Winter, CEO of Avant Technologies. "By partnering with SGAustria, we're ensuring that any genetically modified insulin-producing cells that we develop together can thrive in the body long-term and offer the potential of restoring natural glucose control and dramatically improving patients' quality of life. This technology not only minimizes risks like immune rejection but also prevents potential complications such as cell escape or tumor formation, making it a cornerstone for safe and scalable diabetes therapies."

The diabetes market represents a substantial opportunity. According to the International Diabetes Federation, 589 million people globally live with type 1 and insulin-dependent type 2 diabetes, projected to reach 853 million by 2050. SGAustria's Cell-in-a-Box^® technology creates a protective barrier around genetically modified cells, shielding them from any immune response while allowing nutrients, oxygen, and insulin to pass through freely.

Avant's second joint venture, Klothonova, partners with Singapore-based Austrianova to develop anti-aging therapies and treatments for age-related diseases that restore circulating α-Klotho levels using genetically modified human cells that overexpress the Klotho protein. Research from the Mayo Clinic shows declining α-Klotho levels are associated with arterial stiffness, endothelial dysfunction, and vascular calcification.

Both joint venture projects will utilize cell-encapsulation platforms backed by over 50 publications representing decades of development. The potential market spans multiple therapeutic areas: the global Alzheimer's market is projected to reach $32.8 billion by 2033, cardiovascular disease remains the world's leading cause of death, and kidney disease affects 850 million people worldwide.

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Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) presented first-ever data on CASGEVY in children ages 5-11 years with severe sickle cell disease or transfusion-dependent beta thalassemia at the American Society of Hematology Annual Meeting. In the Phase 3 CLIMB-151 study for SCD, all four patients with sufficient follow-up achieved freedom from vaso-occlusive crises for at least 12 consecutive months, with the longest VOC-free duration approaching two years. In the Phase 3 CLIMB-141 study for TDT, all six evaluable patients achieved transfusion independence for at least 12 months.

"These results — the first clinical data ever presented on any genetic therapy for children ages 5-11 years with SCD — again demonstrate the transformative potential of CASGEVY," said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. "With dosing completed in the 5-11 age group and the Commissioner's National Priority Voucher for CASGEVY in this population in hand, we are excited to begin global regulatory filings in the first half of next year."

Vertex expects to initiate global regulatory submissions for the 5-11 age group in 1H 2026, with the Priority Voucher potentially accelerating review to 1-2 months.

CRISPR Therapeutics (NASDAQ: CRSP) announced positive Phase 1 data from its ongoing clinical trial evaluating CTX310, an investigational in vivo CRISPR/Cas9 gene-editing therapy targeting ANGPTL3, demonstrating dose-dependent, durable reductions in circulating ANGPTL3 and lipids following a single-course intravenous infusion. At the highest dose, patients achieved mean reductions of 73% in ANGPTL3 (maximum 89%), 55% in triglycerides (maximum 84%), and 49% in LDL cholesterol (maximum 87%). Among patients with elevated baseline triglycerides exceeding 150 mg/dL, mean reductions of 60% were observed at therapeutic doses.

"For the first time, we've shown that a single-course in vivo CRISPR treatment can safely and durably lower ANGPTL3, leading to clinically meaningful reductions in triglycerides and LDL," said Naimish Patel, M.D., Chief Medical Officer of CRISPR Therapeutics. "These data provide strong support for continued advancement of CTX310 and our broader cardiovascular gene-editing portfolio."

CTX310 was well tolerated with no treatment-related serious adverse events and no Grade 3 or higher changes in liver transaminases, and CRISPR Therapeutics is advancing the therapy into Phase 1b clinical trials prioritizing severe hypertriglyceridemia and mixed dyslipidemia.

Madrigal Pharmaceuticals, Inc. (NASDAQ: MDGL) presented new two-year data demonstrating Rezdiffra (resmetirom) significantly improved liver stiffness, fibrosis biomarkers, and markers of clinically significant portal hypertension risk in patients with compensated MASH cirrhosis at the American Association for the Study of Liver Diseases meeting. In a new analysis examining patients with more advanced compensated MASH cirrhosis (platelet count <100,000/µL), Rezdiffra demonstrated improvements across multiple imaging tests and biomarkers. Patients experienced mean reductions in liver stiffness of -7.9 kPa and -6.4 kPa respectively, with two thirds shifting to lower Baveno CSPH risk scores by year two.

"MASH cirrhosis carries a 42 times higher risk of liver-related mortality, so there is an urgent need for an approved therapy that can protect patients from progressing to adverse outcomes," said David Soergel, M.D., Chief Medical Officer of Madrigal Pharmaceuticals. "Madrigal is determined to pioneer treatment in compensated MASH cirrhosis, and we are currently executing a fully enrolled Phase 3 outcomes study in this population."

The company's Phase 3 MAESTRO-NASH OUTCOMES trial is evaluating Rezdiffra in compensated MASH cirrhosis, with Rezdiffra currently approved in the U.S. and Europe for MASH with moderate to advanced fibrosis.

Prime Medicine (NASDAQ: PRME) announced the New England Journal of Medicine publication of Phase 1/2 clinical data for PM359, its investigational autologous hematopoietic stem cell product for p47phox chronic granulomatous disease, demonstrating the first-in-human safety and efficacy of Prime Editing technology. Both patients achieved rapid neutrophil engraftment with 69% and 83% dihydrorhodamine-positive neutrophils by Day 30, far exceeding the 20% threshold for clinical benefit, with stable activity suggesting correction in long-term repopulating hematopoietic stem cells. Both patients remained free of new CGD-related complications, with one stopping mesalamine treatment without disease flare.

"Publication of these first-in-human data highlights Prime Editing's promise as a next-generation therapeutic platform, which is capable of delivering meaningful benefits to patients and which can be manufactured and delivered at clinical scale," said Mohammed Asmal, M.D., Ph.D., Chief Medical Officer of Prime Medicine. "Beyond demonstrating early clinical efficacy, these results offer important insights into Prime Editing's safety profile and potential advantages over other gene editing technologies."

No clinically significant adverse events were attributable to PM359, with all observed toxicities consistent with busulfan-based conditioning. The results support Prime Editing's mechanism, which does not induce double-strand breaks, as potentially better tolerated by hematopoietic stem cells than other gene editing approaches.

Source: https://equity-insider.com/2025/03/21/unlocking-the-trillion-dollar-ai-market-what-investors-need-to-know/

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