On February 12, 2026 CRISPR Therapeutics (Nasdaq: CRSP) reported financial results for the fourth quarter and full year ended December 31, 2025.

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"As we close out the fourth quarter, CRISPR Therapeutics continues to make steady progress across a broad and increasingly mature pipeline," said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics. "We made meaningful advances across multiple clinical and preclinical programs, including encouraging data from zugo-cel in autoimmune disease and oncology, continued global uptake of CASGEVY, and important developments across our in vivo liver editing portfolio, and momentum in our siRNA collaboration with Sirius Therapeutics. At the same time, we continue to strengthen our platform capabilities to support long-term value creation. Together, these developments reflect continued execution across the portfolio."

Recent Highlights and Outlook

Hemoglobinopathies and CASGEVY (exagamglogene autotemcel [exa-cel])

CASGEVY is approved in the U.S., EU, Great Britain, Canada, Switzerland, the Kingdom of Saudi Arabia (KSA), the Kingdom of Bahrain, Qatar, the United Arab Emirates (UAE), and Kuwait for patients 12 years and older with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT).
CASGEVY generated fourth quarter 2025 revenue of $54 million and full year 2025 revenue of $116 million, reflecting continued growth in treated patients. 64 patients received infusions of CASGEVY during the year, including 30 in the fourth quarter. Globally, 147 people with SCD or TDT initiated the treatment process with their first cell collection during the year.
Patient initiations and first cell collections increased nearly three-fold in 2025 compared to 2024, with continued momentum going into 2026.
Access to CASGEVY continued to expand across key markets. As of year-end, approximately 90% of patients in the U.S. have reimbursed access to CASGEVY. CASGEVY is also reimbursed in the U.K., Italy, Austria, Denmark, Luxembourg, KSA, the Kingdom of Bahrain, the UAE, and Kuwait. Most recently, in January, Vertex secured reimbursed access for eligible patients with SCD in Scotland, consistent with the prior reimbursement agreement reached in 2025 for patients with TDT.
At the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December 2025, positive pediatric data from the pivotal studies in children ages 5-11 years with SCD or TDT were presented and featured in the "Best of ASH (Free ASH Whitepaper)" program. Global regulatory submissions in this age group are expected to begin in the first half of 2026. The U.S. Food and Drug Administration (FDA) awarded a Commissioner’s National Priority Voucher for this pediatric submission, supporting an expedited review once submissions are complete.
CRISPR Therapeutics continues to advance its in vivo hematopoietic stem cell editing approach utilizing lipid nanoparticle (LNP)-mediated delivery through preclinical studies. This initiative has the potential to expand the addressable patient populations for SCD and TDT.

In Vivo Liver Editing

CRISPR Therapeutics continues to advance a diversified portfolio of in vivo gene editing programs leveraging its proprietary LNP delivery platform.

The Company continues to advance CTX310, targeting angiopoietin-related protein 3 (ANGPTL3), in Phase 1b clinical trials, prioritizing development in severe hypertriglyceridemia (sHTG) and refractory hypercholesterolemia. The Company expects to provide an update in the second half of 2026.
Building on insights from CTX320, the Company is advancing its next-generation LPA program, CTX321. CTX321 incorporates an updated guide RNA that demonstrates approximately two-fold greater potency in preclinical testing while utilizing the same LNP delivery system. CTX321 is currently in IND/CTA-enabling studies, with an Lp(a) program update expected in 2026.
CRISPR Therapeutics continues to advance several preclinical in vivo gene editing candidates, including:

CTX460, targeting SERPINA1 for the treatment of alpha-1 antitrypsin deficiency (AATD), is the first investigational candidate to emerge from the Company’s SyNTase editing platform. The Company expects to initiate a clinical trial for CTX460 in mid-2026.
CTX340, targeting angiotensinogen (AGT) for refractory hypertension, is currently in IND/CTA-enabling studies. The Company expects to initiate a clinical trial for CTX340 in the first half of 2026.

siRNA-based Programs

CRISPR Therapeutics’ small interfering RNA (siRNA)-based portfolio includes clinical-stage programs in cardiovascular and thromboembolic diseases, developed in collaboration with Sirius Therapeutics.

CTX611 (SRSD107), a long-acting siRNA targeting Factor XI (FXI), is in an ongoing Phase 2 clinical trial in patients undergoing total knee arthroplasty (TKA). The Company expects to provide an update in the second half of 2026.
The program has the potential to target a range of thromboembolic and clotting-related indications and represents a multi-billion-dollar market opportunity, including atrial fibrillation (AF), venous thromboembolism (VTE), ischemic stroke, cancer-associated thrombosis (CAT), thrombosis in chronic kidney disease (CKD), peripheral vascular disease (PVD), chronic coronary artery disease (CAD). CRISPR Therapeutics is expected to lead global Phase 3 development of CTX611 with Sirius Therapeutics responsible for development activities in greater China.
CRISPR Therapeutics has the option to nominate up to two siRNA targets for research and development and expects to provide an update in 2026. For each target, CRISPR Therapeutics will fund research and retain opt-in rights to lead clinical development and commercialization. Sirius will be eligible to receive milestone payments and contingent payments, as well as tiered royalties.

Autoimmune Disease and Immuno-Oncology

Zugocabtagene geleucel (zugo-cel; formerly CTX112) continues to advance in both autoimmune disease and hematologic malignancies.

In autoimmune disease, Phase 1 clinical trials of zugo-cel are ongoing across multiple indications, including systemic lupus erythematosus (SLE), systemic sclerosis (SSc), and inflammatory myositis and a second Phase 1 trial in immune thrombocytopenia purpura (ITP) and warm autoimmune hemolytic anemia (wAIHA), with continued progress across studies. The Company expects to provide updates in the second half of 2026.
In immuno-oncology, the Phase 1/2 clinical trial of zugo-cel in B-cell malignancies is ongoing. The Company expects to provide updates in the second half of 2026. Zugo-cel is also being evaluated in combination with pirtobrutinib in aggressive B-cell lymphomas under an existing collaboration with Lilly.
CRISPR Therapeutics continues to leverage its proprietary lipid nanoparticle (LNP) delivery platform, mRNA, and conjugation capabilities to advance its in vivo CAR-T platform with potential applications in autoimmune disease and oncology.
CRISPR Therapeutics’ autoimmune disease and immuno-oncology platforms are supported by a wholly-owned, GMP manufacturing facility located in Framingham, Massachusetts, which provides end-to-end production capabilities for its cell therapy portfolio and supports both clinical and future commercial supply.

Regenerative Medicine

CRISPR Therapeutics continues to advance its regenerative medicine portfolio, including its efforts in diabetes. The Company is advancing CTX213, a deviceless beta cell replacement product candidate consisting of unencapsulated precursor islet cells derived from edited induced pluripotent stem cells (iPSCs), for the treatment of Type 1 diabetes. CTX213 has demonstrated compelling preclinical efficacy via direct administration and is progressing towards the clinic. The Company expects to provide additional updates as development progresses.

Upcoming Events

The Company will present at the upcoming events:

Citi’s 2026 Virtual Oncology Leadership Summit

Date: Wednesday, February 18, 2026

Time: 10:45 a.m. ET

TD Cowen 46th Annual Health Care Conference

Date: Monday, March 2, 2026

Time: 11:10 a.m. ET

Leerink Partners Global Healthcare Conference

Date: Wednesday, March 11, 2026

Time: 1:00 p.m. ET

Fourth Quarter and Full Year 2025 Financial Results

Cash Position: Cash, cash equivalents, and marketable securities were $1,975.8 million as of December 31, 2025, compared to $1,903.8 million as of December 31, 2024. The increase in cash was primarily driven by proceeds from the issuance of common shares, option exercise activity and interest income, offset by operating expenses.
R&D Expenses: R&D expenses were $83.5 million for the fourth quarter of 2025, compared to $71.7 million for the fourth quarter of 2024. The increase in R&D expense was primarily driven by an increase in licensing fees.
G&A Expenses: General and administrative expenses were $18.4 million for the fourth quarter of 2025, compared to $18.1 million for the fourth quarter of 2024.
Collaboration Expense: Collaboration expense, net, was $53.7 million for the fourth quarter of 2025, compared to $10.4 million for the same period in 2024. In 2024, we exercised our option to defer specified costs under the CASGEVY program in excess of the deferral limit of $110.3 million under the amended collaboration agreement with Vertex. The year-over-year increase in collaboration expense reflects the absence of a comparable deferral in 2025.
Net Loss: Net loss was $130.6 million for the fourth quarter of 2025, compared to a net loss of $37.3 million for the fourth quarter of 2024.

About CASGEVY (exagamglogene autotemcel [exa-cel])

CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved for eligible SCD and TDT patients 12 years and older by multiple regulatory bodies around the world.

(Press release, CRISPR Therapeutics, FEB 12, 2026, View Source [SID1234662637])

On February 12, 2026 CorMedix Therapeutics (Nasdaq: CRMD), a biopharmaceutical company focused on developing and commercializing therapeutic products for life-threatening diseases and conditions, reported that senior management will be presenting and participating in investor meetings at upcoming investor conferences taking place in March.

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Leerink Partners Global Healthcare Conference
Date: March 10, 2026
Time: 1:00pm Eastern
Format: Fireside Chat
Webcast: Link to webcast

Citizens Life Sciences Conference
Date: March 11, 2026
Time: 9:35am Eastern
Format: Fireside Chat
Webcast: Link to webcast

Barclays 28th Annual Global Healthcare Conference
Date: March 12, 2026
Format: One-on-one investor meetings

(Press release, CorMedix, FEB 12, 2026, View Source [SID1234662636])

On February 12, 2026 AdvanCell, a clinical-stage radiopharmaceutical company developing innovative targeted alpha therapies for cancer, and 48Hour Discovery, a peptide discovery platform company and GlycoNet-supported startup, reported a collaboration and exclusive licensing agreement to develop a novel peptide-based Lead-212 (²¹²Pb) radioligand therapy program for oncology.

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The collaboration is focused on the development of a purpose-built ²¹²Pb-targeted radioligand therapy, including a peptide ligand optimized for delivery of AdvanCell’s lead-212 (²¹²Pb) alpha-emitting payload. While centered on a 212Pb targeted RLT, the work also serves as a proof point for the broader applicability of 48Hour Discovery’s peptide discovery engine in radioligand therapy (RLT) development, demonstrating its ability to generate ligands compatible with targeted alpha delivery.

Precision Alpha Therapy, Enabled by Rapid Peptide Discovery
Lead-212 is a potent alpha-emitting radionuclide that enables localized, high-energy radiation delivery while minimizing damage to surrounding healthy tissue. AdvanCell has built an integrated platform around ²¹²Pb spanning isotope supply, manufacturing, and clinical development.

48Hour Discovery’s platform—combining large phage display libraries, high-throughput screening, and AI and chemical optimization—has been designed to rapidly generate peptides with high affinity and specificity. This program highlights the platform’s capacity to meet the stringent requirements of RLT development, including selectivity, PK and biodistribution properties, stability and payload compatibility.

"This collaboration reflects the strength of our ²¹²Pb platform and our ambition to translate scientific innovation into novel targeted alpha therapies that can meaningfully change outcomes for patients," said Philina Lee, PhD, CEO of AdvanCell. "It also demonstrates how innovative discovery technologies can be applied to accelerate the development of highly targeted alpha therapies."

Rick Finnegan, CEO of 48Hour Discovery, added:
"We see this program as an important validation of our discovery engine in the radioligand therapy space. It underscores the broader utility of our platform for generating ligands suited to demanding oncology applications. Importantly, this agreement is one of several partnerships we have entered into recently with companies developing radioligand therapies, and together they highlight the versatility of our platform across this rapidly growing modality."

(Press release, GlycoNet, FEB 12, 2026, View Source;utm_medium=rss&utm_campaign=advancell-and-glyconet-supported-startup-48hour-discovery-announce-collaboration-and-exclusive-licensing-agreement [SID1234662635])

On February 12, 2026 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) ("BioMarin") reported that it closed its previously announced offering of $850 million of 5.500% senior unsecured notes due 2034 (the "Notes"). The issue price of the Notes is 100.000%.

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BioMarin intends to use the net proceeds from the offering of the Notes, together with borrowings under a new $2 billion senior secured term loan "B" facility (the "Term Loan B Facility") and $800 million senior secured term loan "A" facility (the "Term Loan A Facility" and, together with the Term Loan B Facility, the "Term Facilities") and cash on hand, to fund the consideration payable in connection with the pending acquisition (the "Acquisition") of Amicus Therapeutics, Inc. ("Amicus") and related fees and expenses in connection with the Acquisition and borrowings under the Term Facilities and the offering of the Notes. In addition to the Term Facilities, BioMarin expects to enter into a $600 million senior secured revolving credit facility connection with the Acquisition (the "New Revolving Facility" and, together with the Term Facilities, the "New Senior Secured Credit Facilities"). BioMarin may borrow up to $150 million under the New Revolving Facility to pay such fees and expenses.

Gross proceeds from the offering of the Notes were deposited into an escrow account at the closing of the offering, pending consummation of the Acquisition. In the event that the Acquisition is not completed on or prior to December 19, 2026, or upon the occurrence of certain other events, BioMarin will be required to redeem all of the Notes at a redemption price equal to 100% of the initial issue price of the Notes plus accrued and unpaid interest from the date of issuance, or the most recent date to which interest has been paid or provided for, to but excluding the special mandatory redemption date.

The Notes are jointly and severally guaranteed by certain of BioMarin’s subsidiaries that will guarantee the obligations under the New Senior Secured Credit Facilities, including, after the closing of the Acquisition, Amicus and certain of its subsidiaries that will guarantee the obligations under the New Senior Secured Credit Facilities.

The indenture governing the Notes contains customary covenants that, among other things, restrict, with certain exceptions, the ability of each of BioMarin and its subsidiaries to incur additional debt, pay dividends, make certain other restricted payments, incur debt secured by liens, dispose of assets, engage in consolidations and mergers or sell or transfer all or substantially all of its assets.

The Notes have not been, and will not be, registered under the Securities Act of 1933, as amended (the "Securities Act"), or any state or other securities laws and may not be offered or sold in the United States absent an effective registration statement or an applicable exemption from the registration requirements of or in a transaction not subject to the Securities Act and any state or other applicable securities laws. Accordingly, the Notes were offered and sold only to a limited number of persons who were either (1) reasonably believed to be "qualified institutional buyers" as defined in Rule 144A under the Securities Act or (2) non-U.S. persons outside the United States pursuant to Regulation S under the Securities Act. The Notes are subject to restrictions on transferability and resale and may not be transferred or resold except in compliance with the registration requirements of the Securities Act or pursuant to an exemption therefrom and in compliance with any state or other applicable securities laws.

This press release is for information purposes only and is not intended to and does not constitute, or form part of, an offer, invitation or the solicitation of an offer or invitation to purchase, otherwise acquire, subscribe for, sell or otherwise dispose of any securities, or the solicitation of any vote or approval in any jurisdiction, pursuant to the proposed transaction or otherwise, nor shall there be any sale, issuance or transfer of securities in any jurisdiction in contravention of applicable law.

(Press release, BioMarin, FEB 12, 2026, View Source [SID1234662634])

On February 12, 2026 Arvinas, Inc. (Nasdaq: ARVN), a clinical-stage biotechnology company creating a new class of drugs based on targeted protein degradation, reported the appointment of Randy Teel, Ph.D., as President and Chief Executive Officer (CEO), as well as a member of the Company’s Board of Directors, effective today. Dr. Teel previously served as Arvinas’ Chief Business Officer (CBO) and is recognized for his strategic leadership in advancing corporate growth and fostering strong industry partnerships.

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Dr. Teel succeeds John Houston, Ph.D., who is retiring from his role as President, Chief Executive Officer, and Chair of Arvinas’ Board of Directors. Dr. Houston will continue to serve as a member of the Board and has entered into a consulting agreement with Arvinas whereby he will provide consulting and advisory services to the company. Briggs Morrison, M.D., has been elected to serve as Chair of the Arvinas Board of Directors, effective today.

"After a comprehensive search to identify the next leader for Arvinas, we are pleased to announce Randy as President and Chief Executive Officer," said Briggs Morrison, M.D., Chair, Arvinas Board of Directors. "Randy brings a strategic vision, a deep understanding of the company’s scientific and operational foundation, and an ability to understand and address the priorities of multiple stakeholders. The Board was impressed with Randy’s role in refocusing Arvinas’ strategy around its differentiated and exciting clinical pipeline, and his strategic experience across the biotechnology industry uniquely positions him to lead Arvinas through its next phase of growth. On behalf of the Board, I extend our sincere thanks to John for his exceptional leadership, vision, and contributions over the past nine years."

Dr. Teel joined Arvinas in 2018 and brings 20 years of biopharmaceutical industry experience with a strong track record of impact. Dr. Teel played a critical role in Arvinas’ 2018 initial public offering (IPO) and the company’s transition to a public, clinical-stage biotechnology company. As CBO, Dr. Teel was responsible for corporate strategy, business development, investor relations, and communications, as well as leadership roles driving strategic initiatives and long-range planning. In earlier roles at Arvinas, Dr. Teel led commercial development and served as Arvinas’ interim Chief Financial Officer and Treasurer. Dr. Teel formerly served as Vice President and Head of Strategy at Alexion Pharmaceuticals, where he led long-range planning and partnered with R&D and commercial teams to shape inline and lifecycle management strategies. Prior to Alexion, Dr. Teel was an Associate Partner at McKinsey & Company, where he advised biopharmaceutical clients on issues in commercial, medical, and development. He holds a B.Sc. in Biology from Gonzaga University and a Ph.D. in Immunobiology from Yale University.

"I am honored to step into this role at such a pivotal moment for Arvinas and to build on the strong foundation and momentum we’ve created," said Dr. Teel. "Following our first-ever successful pivotal trial of a PROTAC degrader, we’re directing our attention to our earlier stage clinical programs and their potential to transform treatment paradigms for patients with serious diseases. Under John’s leadership, we have created the leading PROTAC platform in the industry, consistently generating high-quality candidates and advancing programs with the potential to become transformative treatments for patients. I look forward to continuing this work with the exceptional team at Arvinas as we advance our mission and deliver meaningful impact for patients, their families, and our shareholders."

During his tenure as President and CEO, Dr. Houston advanced Arvinas through major milestones, including progressing multiple investigational programs into clinical development, reporting the first positive pivotal results for a PROTAC degrader, and demonstrating central nervous system pharmacodynamic activity for an orally administered PROTAC. Dr. Houston led Arvinas to its 2018 IPO and raised over $2 billion through private funding, partnerships and strategic collaborations, and follow-on financings. These achievements have established Arvinas as a leader in targeted protein degradation and positioned the Company for long-term success.

"I’m proud of all we have accomplished together and confident that Randy will lead Arvinas to new levels of innovation and impact," said Dr. Houston. "We have a robust pipeline, and the team is well-positioned under Randy’s leadership as we approach several anticipated critical milestones later this year, including important clinical data from our LRRK2, BCL6 and KRAS G12D degraders. I look forward to supporting Randy and the entire organization as Arvinas continues to push the boundaries of what’s possible for patients."

(Press release, Arvinas, FEB 12, 2026, View Source [SID1234662633])