Precision BioSciences Achieves Clinical Milestone in Collaboration with TG Therapeutics, Securing $7.5 Million in Proceeds from Azer-Cel Program for Multiple Sclerosis
Precision BioSciences, Inc, a leading clinical-stage biotechnology company specializing in gene editing technologies, announced today a significant milestone achievement under its licensing agreement with TG Therapeutics, Inc. (Nasdaq: TGTX). This milestone is linked to the development of azercabtagene zapreleucel (azer-cel), an innovative therapeutic candidate for progressive forms of multiple sclerosis (MS). The achievement marks an important step forward in the development of Precision’s ARCUS®-based in vivo gene editing therapies for diseases with high unmet medical needs.
The milestone event was triggered by progress in the Phase 1 clinical trial of azer-cel, reflecting the continued advancement of the therapy in patients with progressive forms of MS. In recognition of this progress, TG Therapeutics has committed to provide Precision BioSciences with total proceeds of $7.5 million. This includes a cash component of $5.25 million and an additional $2.25 million through the purchase of 201,504 shares of Precision common stock at $11.17 per share, in accordance with the terms of the license agreement.
The financial implications of this milestone are significant for Precision BioSciences. With existing cash and cash equivalents, including the proceeds from the azer-cel milestone, the company expects to maintain a strong cash runway through 2028. This financial stability is further supported by disciplined fiscal management and the availability of its at-the-market (ATM) facility, ensuring sufficient resources to continue advancing its pipeline of in vivo gene editing therapies.
Michael Amoroso, President and Chief Executive Officer of Precision BioSciences, commented on the achievement, saying, “We are pleased with TG Therapeutics’ continued advancement of azer-cel in progressive multiple sclerosis and with the achievement of this clinical milestone. Their clinical progress reflects the potential of azer-cel in autoimmune diseases such as multiple sclerosis and underscores the value of our strategic partnering approach. Importantly, it also highlights the additive value of our partnered programs as we enter a catalyst-rich 2026, with multiple potential data and development milestones complementing progress across our wholly-owned in vivo gene editing pipeline.”
The collaboration between Precision BioSciences and TG Therapeutics stems from a licensing agreement signed in January 2024. Under this agreement, TG Therapeutics obtained exclusive worldwide rights to develop and commercialize azer-cel for autoimmune diseases and other non-oncology indications. Precision BioSciences received an upfront payment and structured potential near-term economics tied to development and commercialization milestones. The company remains eligible to receive additional payments contingent upon achieving specific development, regulatory, and commercial milestones, as well as royalties on net sales. Collectively, these future milestone payments could total up to $288 million, with royalties ranging from high single-digit to low double-digit percentages of net sales.
Azer-cel represents an important innovation in the field of autoimmune disease therapeutics. Developed using Precision’s proprietary ARCUS® genome editing platform, azer-cel is designed to target specific disease pathways by precisely editing genes within the patient’s own cells. The ARCUS platform leverages a unique engineered nuclease derived from a naturally occurring homing endonuclease, enabling highly targeted gene editing with potentially fewer off-target effects compared to other gene editing technologies. This precision is critical in the treatment of complex autoimmune disorders such as multiple sclerosis, where the immune system attacks the body’s own central nervous system.
The ongoing Phase 1 clinical trial of azer-cel in progressive MS aims to evaluate the safety, tolerability, and preliminary efficacy of the therapy in patients who have limited treatment options. Positive outcomes from this trial could pave the way for further clinical development and eventual regulatory approval, representing a potential paradigm shift in the treatment of progressive MS and other autoimmune conditions.
Beyond azer-cel, Precision BioSciences continues to advance a robust pipeline of in vivo gene editing therapies addressing a broad spectrum of diseases. The company’s proprietary ARCUS technology underpins these efforts, offering the potential for precise and durable therapeutic effects across multiple indications. The successful milestone with TG Therapeutics reinforces the strategic value of Precision’s partnership approach, enabling the company to leverage external expertise and resources while retaining significant upside through milestone payments and royalties.
Financially, the milestone proceeds provide additional liquidity that supports ongoing research and development programs, operational initiatives, and strategic investments. The combination of existing cash reserves, milestone payments, and disciplined financial planning positions Precision BioSciences to execute its growth strategy over the next several years, including the advancement of its wholly-owned pipeline and the pursuit of new partnership opportunities.
Michael Amoroso emphasized the broader implications of the milestone, stating, “This achievement not only reflects the progress of azer-cel in autoimmune diseases but also highlights the broader potential of our partnered programs to generate meaningful clinical and financial value. We are entering 2026 with multiple upcoming catalysts, including potential data readouts and regulatory milestones across both partnered and wholly-owned programs, which together could accelerate the growth and impact of our gene editing portfolio.”
The milestone also demonstrates the growing significance of gene editing technologies in the treatment of diseases with high unmet medical needs. With precision, targeted approaches such as ARCUS, patients may benefit from therapies that address the root cause of disease rather than merely managing symptoms. Precision BioSciences’ continued commitment to innovation in this field underscores its role as a leader in developing next-generation gene editing therapies.
In conclusion, the achievement of the clinical milestone with TG Therapeutics for azer-cel represents a significant validation of Precision BioSciences’ strategic approach, its innovative ARCUS platform, and the potential of gene editing to transform the treatment landscape for autoimmune diseases. The $7.5 million in milestone proceeds, combined with existing resources and disciplined financial management, provides the company with the runway to continue advancing its portfolio of in vivo gene editing therapies and pursue additional clinical, regulatory, and commercial milestones in the coming years. As Precision BioSciences moves forward, the company remains well-positioned to capitalize on both its wholly-owned and partnered programs, driving innovation and delivering potential value to patients, partners, and shareholders alike.
