On February 12, 2025 Ferring Pharmaceuticals reported three abstracts featuring ADSTILADRIN (nadofaragene firadenovec-vncg) will be presented at the 2025 American Society of Clinical Oncologists Genitourinary Cancers (ASCO GU) Symposium held February 13-15 in San Francisco (Press release, Ferring Pharmaceuticals, FEB 12, 2025, View Source [SID1234650228]). ADSTILADRIN is the first and only intravesical non-replicating gene therapy approved by the U.S. Food and Drug Administration (FDA) for patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors (±Ta/T1).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Mayo Clinic will present additional updated early results in a poster presentation of an independent, retrospective real-world outcomes study conducted at three of their sites. The study is evaluating complete response rates, high-grade recurrence-free survival, cystectomy-free survival, overall survival, and adverse events among patients treated with ADSTILADRIN for high-risk BCG-unresponsive NMIBC.

"Understanding real-world outcomes in patients with BCG-unresponsive NMIBC is essential for informing how we approach treatment and deliver care," said Mark D. Tyson, M.D., urologic oncologist and associate professor, Mayo Clinic, Scottsdale, Ariz., and principal investigator of this independent study. "These initial encouraging post-marketing results provide valuable insights into the benefits of using ADSTILADRIN in everyday clinical practice, allowing providers to make more informed decisions and deliver the best possible care for patients."

Additional abstracts to be presented include the study protocols for two studies in the ABLE (ADSTILADRIN in BLadder CancEr) clinical trial program – the three-arm ABLE-22 study and the two-arm ABLE-32 study. Clinical sites were recently activated for both studies which are investigating the efficacy and safety of ADSTILADRIN in high-risk and intermediate-risk NMIBC.

"These real-world data extend the body of evidence for efficacy, safety, and low treatment burden of ADSTILADRIN in NMIBC patients who no longer respond to BCG, building on the positive five-year results from our pivotal Phase 3 trial announced last year," said Bipin Dalmia, Global Head of Uro-Oncology & Urology Franchise, Ferring Pharmaceuticals. "As the first and only FDA approved intravesical bladder-sparing monotherapy for BCG-unresponsive NMIBC patients, ADSTILADRIN is fully available throughout the United States, offering a convenient quarterly dosing schedule that may help encourage treatment adherence. The continuation of our clinical trial program further highlights our commitment to advancing the science in our journey to establish ADSTILADRIN as the new standard of care and backbone therapy across the urothelial cancer disease spectrum."

About ADSTILADRIN Presentations at ASCO (Free ASCO Whitepaper) GU
ADSTILADRIN poster titles and presentation times at ASCO (Free ASCO Whitepaper) GU, February 13-15, 2025, are:

Real-world outcomes of nadofaragene firadenovec in BCG-unresponsive non-muscle invasive bladder cancer. Abstract #716 (Poster #D13), Friday, February 14, 11:30 a.m. PST
ABLE-22: Safety and efficacy evaluation of nadofaragene firadenovec alone or in combination with chemotherapy or immunotherapy—a randomized, open-label, phase 2 study. Abstract #TPS891 (Poster #J19), Friday, February 14, 11:30 a.m. PST
ABLE-32: A randomized, controlled, phase 3B clinical trial of nadofaragene firadenovec-vncg versus observation in patients with intermediate-risk non–muscle-invasive bladder cancer. Abstract #TPS888 (Poster #J16), Friday, February 14, 11:30 a.m. PST
About ABLE-22 and ABLE-32
ABLE-22 (NCT06545955) is a three-arm, Phase 2 trial evaluating ADSTILADRIN as a monotherapy and as part of combination with chemotherapy or an immune checkpoint inhibitor in patients with high-risk BCG-unresponsive NMIBC. Investigators in all three arms of the ABLE-22 study will have the option to re-induce appropriate patients who do not achieve a complete response to the initial single dose or combination regimen, an option that was not included in the original Phase 3 study.

The Phase 3B ABLE-32 (NCT06510374) trial is assessing ADSTILADRIN in patients with intermediate-risk NMIBC (IR NMIBC), for which there are no U.S. FDA-approved treatment options.

About ADSTILADRIN
ADSTILADRIN (nadofaragene firadenovec-vncg) is the first and only FDA-approved intravesical non-replicating gene-therapy for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. It is a non-replicating adenovirus vector-based therapy containing the gene interferon alfa-2b, administered locally as a monotherapy by catheter directly into the bladder once every three months. The vector enters the cells of the bladder wall, releasing the active gene and causing the bladder’s cell walls to secrete high and transient local expression of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer. This approach essentially turns the bladder wall cells into interferon microfactories, enhancing the body’s own natural defenses against the cancer.

ADSTILADRIN has been studied in a clinical trial program that includes 157 patients with high-risk, BCG-unresponsive NMIBC who had been treated with adequate BCG previously and did not see benefit from additional BCG treatment (full inclusion criteria published on clinicaltrials.gov: NCT02773849).1

Ferring is leading the future in uro-oncology treatment with ADSTILADRIN at the center, while expanding access with the support of new, state-of-the-art manufacturing facilities. As announced in January 2024, ADSTILADRIN is fully available and accessible in the U.S. ADSTILADRIN has confirmed 99 percent coverage for commercial and government-insured patients. As of April 1, 2024, in accordance with the Centers for Medicare and Medicaid Services (CMS), ADSTILADRIN established an Average Sales Price (ASP). Since the establishment of ASP, all covered claims submitted for reimbursement have received payment within an average of 25 days.2

Ferring is committed to investing in novel therapies, developing life-changing solutions that address unmet medical needs, and aiding the uro-oncology community in helping patients live better lives. More information is available in the U.S. at FerringUroOncology.com and on the dedicated Ferring Uro-Oncology channels on LinkedIn and X.

About Non-Muscle Invasive Bladder Cancer (NMIBC)
NMIBC is a form of bladder cancer that is found in the inner layer cells of the bladder and does not invade into or beyond the muscle wall.3 In the United States, bladder cancer is the sixth most common cancer,4 fourth among men,5 and it is estimated that there will be approximately 84,870 new cases of bladder cancer in the U.S. in 2025.5 Historically, 75% of bladder cancer presents as NMIBC.6 In patients with high-risk NMIBC, intravesical BCG remains the first-line standard-of-care. However, approximately one third of patients with NMIBC will not respond to BCG therapy and 50% of those with an initial response will experience recurrence or progression of their disease.7 Current treatment options for BCG-unresponsive patients are very limited, and National Comprehensive Cancer Network (NCCN) guidelines recommend cystectomy (partial or complete removal of the bladder).8

INDICATION
ADSTILADRIN is a non-replicating adenoviral vector-based gene therapy indicated for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS: ADSTILADRIN is contraindicated in patients with prior hypersensitivity reactions to interferon alfa or to any component of the product.

WARNINGS AND PRECAUTIONS:

Risk with delayed cystectomy: Delaying cystectomy in patients with BCG-unresponsive CIS could lead to development of muscle invasive or metastatic bladder cancer, which can be lethal. If patients with CIS do not have a complete response to treatment after 3 months or if CIS recurs, consider cystectomy.
Risk of disseminated adenovirus infection: Persons who are immunocompromised or immunodeficient may be at risk for disseminated infection from ADSTILADRIN due to low levels of replication-competent adenovirus. Avoid ADSTILADRIN exposure to immunocompromised or immunodeficient individuals.
DOSAGE AND ADMINISTRATION: Administer ADSTILADRIN by intravesical instillation only. ADSTILADRIN is not for intravenous use, topical use, or oral administration.

USE IN SPECIFIC POPULATIONS: Advise females of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 6 months after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 3 months after the last dose.

ADVERSE REACTIONS: The most common (>10%) adverse reactions, including laboratory abnormalities (>15%), were glucose increased, instillation site discharge, triglycerides increased, fatigue, bladder spasm, micturition (urination urgency), creatinine increased, hematuria (blood in urine), phosphate decreased, chills, pyrexia (fever), and dysuria (painful urination).

You are encouraged to report negative side effects of prescription drugs to FDA. Visit www.FDA.gov/medwatch or call 1-800-332-1088. You may also contact Ferring Pharmaceuticals at 1-888-FERRING.

Please click to see the full Prescribing Information.

On February 12, 2025 Replimune Group, Inc. (Nasdaq: REPL), a clinical stage biotechnology company pioneering the development of novel oncolytic immunotherapies, reported financial results for the fiscal third quarter ended December 31, 2024 and provided a business update (Press release, Replimune, FEB 12, 2025, View Source [SID1234650211]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"Over the past couple of months, we have achieved significant regulatory milestones for RP1 in anti-PD-1 failed melanoma," said Sushil Patel, Ph.D., CEO of Replimune. "With Priority Review and a PDUFA date set for July 22, 2025, by the FDA, our efforts are focused on ensuring a successful commercial launch of RP1 upon approval. Our commercial strategy is built on a deep understanding of the patient population and prescriber landscape, coupled with a launch model designed to effectively deliver intratumoral therapy. With over $500 million in cash, we are well-capitalized to execute our plans and are excited to provide further updates as we transition to a commercial-stage company."

Program Highlights & Milestones

RP1

RP1 combined with Opdivo (nivolumab) in anti-PD1 failed melanoma
In January, the FDA accepted the BLA for RP1 in combination with nivolumab for patients with advanced melanoma. The BLA was granted Priority Review by the FDA with a PDUFA action date of July 22, 2025.
The BLA is supported by the primary analysis data of the IGNYTE trial, evaluating RP1 combined with nivolumab in patients with anti-PD-1 failed melanoma.
Enrolling into the confirmatory Phase 3 trial, IGNYTE-3, with over 100 sites planned globally. This trial will assess RP1 in combination with nivolumab in patients with advanced melanoma who have progressed on anti-PD-1 and anti-CTLA-4 therapies or are ineligible for anti-CTLA-4 treatment.
RP2

RP2 in uveal melanoma
Enrolled the first patient in a registration-directed study of RP2 in metastatic uveal melanoma in patients who are immune checkpoint inhibitor-naïve. The study will enroll approximately 280 patients and evaluate RP2 in combination with nivolumab versus ipilimumab in combination with nivolumab. The primary endpoints of the study are overall survival and progression free survival and key secondary endpoints are overall response rate and disease control rate.
RP2 in hepatocellular carcinoma (HCC)
Enrolled the first patient in a Phase 2 clinical trial with RP2 combined with atezolizumab and bevacizumab in anti-PD1/PD-L1 progressed HCC. The trial is an open label trial that will enroll 30 patients and evaluate RP2 combined with the second-line therapy of atezolizumab and bevacizumab. The study is being conducted under a collaboration and supply agreement with Roche.
Financial Highlights

Financing: Completed a public offering of shares of the Company’s common stock and pre-funded warrants, raising approximately $156.0 million net of issuance costs. Proceeds from the financing will be used to fund the continued development of our RPx platform, including expanding our ongoing studies within RP1 and broadening clinical development plans for RP2, as well as for working capital and general corporate purposes.
Cash Position: As of December 31, 2024, cash, cash equivalents and short-term investments were $536.5 million, as compared to $420.7 million as of fiscal year ended March 31, 2024. The increase in cash balance was directly related to the public offering in November, somewhat offset by cash utilized in operating activities in advancing the Company’s clinical development plans.

Based on the current operating plan, the Company believes that existing cash, cash equivalents and short-term investments, as of December 31, 2024 will enable the Company to fund operations into the fourth quarter of 2026 which includes scale up for the potential commercialization of RP1 in skin cancers and for working capital and general corporate purposes and excludes any potential revenue.
R&D Expenses: Research and development expenses were $48.0 million for the fiscal third quarter ended December 31, 2024, as compared to $42.8 million for the fiscal third quarter ended December 31, 2023. This increase was primarily due to an increase in personnel-related costs, as well as consulting and facility-related costs. Research and development expenses included $4.6 million in stock-based compensation expenses for the fiscal third quarter ended December 31, 2024.
S,G&A Expenses: Selling, general and administrative expenses were $18.0 million for the fiscal third quarter ended December 31, 2024, as compared to $13.7 million for the fiscal third quarter ended December 31, 2023. Selling, general and administrative expenses included $4.1 million in stock-based compensation expenses for the fiscal third quarter ended December 31, 2024.
Net Loss: Net loss was $66.3 million for the fiscal third quarter ended December 31, 2024, as compared to a net loss of $51.1 million for the fiscal third quarter ended December 31, 2023.
About RP1

RP1 (vusolimogene oderparepvec) is Replimune’s lead product candidate and is based on a proprietary strain of herpes simplex virus engineered and genetically armed with a fusogenic protein (GALV-GP R-) and GM-CSF intended to maximize tumor killing potency, the immunogenicity of tumor cell death, and the activation of a systemic anti-tumor immune response.

About RP2

RP2 is based on a proprietary strain of herpes simplex virus engineered and genetically armed with a fusogenic protein (GALV-GP R-) and GM-CSF intended to maximize tumor killing potency, the immunogenicity of tumor cell death and the activation of a systemic anti-tumor immune response. RP2 additionally expresses an anti-CTLA-4 antibody-like molecule, as well as GALV-GP R- and GM-CSF. RP2 is intended to provide targeted and potent delivery of these proteins to the sites of immune response initiation in the tumor and draining lymph nodes, with the goal of focusing systemic-immune-based efficacy on tumors and limiting off-target toxicity.

On February 12, 2025 HAYA Therapeutics, SA, a biotechnology company pioneering precision RNA-guided regulatory genome targeting therapeutics for rare, common, chronic and age-associated diseases, reported that it has been awarded the prestigious Innosuisse Certificate for Sustainable Growth from Innosuisse, the Swiss Innovation Agency, who also provided funding to support HAYA’s research into using its technology to address cancer (Press release, Haya Therapeutics, FEB 12, 2025, View Source [SID1234650229]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

In addition to Innosuisse’s award for HAYA’s potential for growth and success, it is providing 1.5M CHF (approximately $1.64M USD) in non-dilutive funding for HAYA through Innosuisse’s Start-up Innovation Projects, recognizing HAYA’s cutting-edge approach and broad applicability of their long non-coding RNA (lncRNA) platform in developing disease-modifying therapeutics beyond the heart and into oncology.

The funds will be used over the next two years for translational studies to further develop an innovative therapy targeting cancer-associated fibroblast-specific lncRNA for aggressive solid tumors.

"Solid tumors, which make up approximately 90% of all cancers, require innovative therapeutic strategies due to their complexity and resistance to traditional treatments," said Samir Ounzain, Ph.D., Co-founder and CEO of HAYA Therapeutics. "Fibrosis plays a critical role in shaping the tumor microenvironment, creating a rigid and immunosuppressive barrier that fuels cancer progression and limits treatment efficacy. At HAYA, we are pioneering a novel RNA-guided approach to precisely reprogram cancer-associated fibroblasts, disrupting the fibrotic niche and unlocking new possibilities for more effective and durable cancer therapies."

"By leveraging the exquisite cell specificity of lncRNAs coupled with antisense oligonucleotide’s broad distribution to extra hepatic tissues including tumors, HAYA is able to drug disease modifying targets within the tumor microenvironment. This novel paradigm holds tremendous potential as a next generation treatment strategy for immunotherapy resistant solid tumors such as for pancreatic cancer and triple negative breast cancer," said Sudhir Agrawal, DPhil, FSRC, a member of HAYA’s scientific advisory board and Founder of Arnay Sciences. "HAYA’s strategy could be transformational and offer disease-modifying therapy options to patients in need."

HAYA’s unique RNA-guided regulatory genome technology enables the discovery and development of innovative RNA-based therapeutics for a wide range of diseases. HAYA’s lead candidate, HTX-001, is being developed for the treatment of heart failure, with other preclinical therapeutics in development for other indications.

On February 12, 2025 Rezolute, Inc. (Nasdaq: RZLT) ("Rezolute" or the "Company"), a late-stage biopharmaceutical company dedicated to developing transformative therapies for rare diseases with serious unmet needs, reported financial results and provided a business update for the three months ended December 31, 2024 (Press release, Rezolute, FEB 12, 2025, View Source [SID1234650212]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We have made significant regulatory progress with ersodetug and our focus in 2025 remains squarely on advancing both Phase 3 trials for patients with congenital HI and tumor HI," said Nevan Charles Elam, Chief Executive Officer and Founder of Rezolute. "We are encouraged by our momentum and remain dedicated to providing meaningful and innovative treatments for patients with limited options."

Recent Pipeline Progress and Anticipated Milestones

Congenital HI

· The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation to ersodetug for the treatment of hypoglycemia due to congenital HI.
·
sunRIZE, a Phase 3, multicenter, double-blind, randomized, controlled safety and efficacy registrational study, is ongoing.

○ Enrollment of U.S. participants is anticipated to occur in the second quarter of 2025.
○ Overall study enrollment expected to conclude in the second quarter of 2025, with topline results expected in the fourth quarter of 2025, subject to outcomes from an interim analysis.

· An independent Data Monitoring Committee (DMC) reviewed safety and pharmacokinetics of ersodetug in eight infant participants in the open-label arm of the sunRIZE study.
○ Ersodetug administered between 5-10 mg/kg during a bi-weekly loading phase and a monthly maintenance phase was generally safe and well-tolerated.
○ Observed ersodetug drug levels at peak and trough were comparable to exposures in older pediatric participants in the Phase 2b RIZE study and validate the chosen dose regimen of 5 and 10 mg/kg administered bi-weekly and monthly.
○ Subsequent infant participants may be enrolled into the double-blind, placebo-controlled study.
○ The DMC did not analyze efficacy and the Company remains blinded to the results.

· An interim analysis of the primary study endpoint (change in hypoglycemia events) is planned for this quarter, with results and study update to be announced early in the second quarter.
○ Three possible outcomes from the analysis include: (i) futility and the study should be stopped, (ii) continue the study as is or (iii) continue the study as is but increase the sample size by 33% (18 additional patients) to enhance statistical confidence in the final outcome.

Tumor HI

· During the quarter, FDA granted Orphan Drug Designation to ersodetug for the treatment of hypoglycemia due to tumor HI.
· A Phase 3 registrational study for ersodetug in patients with tumor HI is anticipated to begin in the first half of 2025.
· Topline results are expected in the second half of 2026.

Fiscal Second Quarter Financial Results

Cash, cash equivalents and investments in marketable securities were $105.3 million as of December 31, 2024, compared to $127.1 million as of June 30, 2024.

Research and development expenses were $12.6 million for the second quarter of fiscal 2025, compared with $12.0 million for the same period a year ago, with the increase primarily attributable to increased expenditures in clinical trial activities, manufacturing costs and higher personnel-related expenses, which include employee compensation.

General and administrative expenses were $4.5 million for the second quarter of fiscal 2025, compared with $3.2 million for the same period a year ago, with the increase primarily attributable to professional fees and employee-related expenses as a result of increased headcount.

Net loss was $15.7 million for the second quarter of fiscal 2025 compared with a net loss of $13.9 million for the same period a year ago.

About Ersodetug

Ersodetug is a fully human monoclonal antibody that binds to a unique allosteric site on insulin receptors to counteract the effects of insulin receptor over-activation by insulin and related substances (such as IGF-2), thereby improving hypoglycemia in the setting of hyperinsulinism (HI). Because ersodetug acts downstream from the pancreas, it has the potential to be universally effective at treating hypoglycemia due to any form of HI.

About sunRIZE

The Phase 3 sunRIZE study is a multi-center, randomized, double-blind, placebo-controlled, parallel arm study designed to evaluate the efficacy and safety of ersodetug in patients with congenital HI who are experiencing poorly controlled hypoglycemia. Participants between the ages of 3 months to 45 years old are eligible to participate. The study is enrolling up to 56 participants in more than a dozen countries around the world.

On February 12, 2025 Terrain Biosciences reported the company launches from stealth as the world’s first RNA design-build company, using advanced AI and its proprietary manufacturing platform to accelerate development of the next generation of programmable medicines (Press release, Terrain Biosciences, FEB 12, 2025, View Source [SID1234650230]). RNA-based modalities, including mRNA, cell & gene therapies, and emerging personalized cancer vaccines are promising and rapidly advancing, with hundreds of drug candidates in development for indications ranging from oncology to infectious disease to neurodegenerative illnesses. Despite incredible progress, innovators in the space continue to face challenges in designing, selecting, manufacturing, and delivering optimal customized RNA sequences for therapeutic benefit.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Founded by Professors Omar Abudayyeh and Jonathan Gootenberg of Harvard Medical School and Professor Patrick Hsu of the Arc Institute and University of California, Berkeley, the company is led by industry veterans including CSO Aaron Larsen (formerly at Beam & Moderna), COO Ted Tisch (formerly at Mammoth Biosciences and Synthego), and founding CEO Chetan Tadvalkar (veteran GM/COO of pharma services companies, including former head of business operations at Ginkgo Bioworks). Terrain has raised $9M in seed funding to bring cutting-edge research in RNA design and manufacturing to innovators building RNA-based therapies and vaccines, and support development of personalized cancer vaccines. Over a dozen biotechs already trust Terrain Bio to help them identify and produce sequences with better expression, greater durability, and optimal manufacturability, accelerating their path to the clinic.

"AI is poised to dramatically accelerate how we design and develop drugs, but the reality is that in silico models can only get us so far in RNA — bringing a drug from the lab to the clinic requires bringing advanced science into the real world. Our partners need excellent machine learning tools combined with a real manufacturing platform and data to enable and accelerate their path to the clinic," said Terrain co-founder Omar Abudayyeh.

"Our partners are brilliant scientists on an incredibly challenging journey to develop new medicines and ultimately help patients," said Chetan Tadvalkar, CEO of Terrain. "Our team has been part of similar journeys before, and today our purpose is to help scientists navigate the complexity of designing the right RNA sequence and guide them towards their goal while ensuring they hit key preclinical milestones."

RNA optimization is critical for therapeutic efficacy and safety — sequence and purity reaches beyond the mechanism of action to have broad impacts across expression, stability, durability, immunogenicity, and manufacturability. "The search space for designing RNA is nearly infinite," said co-founder Jonathan Gootenberg. "Navigating that space computationally and experimentally will unlock enormous potential in medicine, but remains incredibly challenging given the lack of top-quality data for training better models."

"Bridging design and manufacturing in a tight feedback loop means higher quality preclinical data, which means stronger generative models for design, which means better drug candidates," said co-founder Patrick Hsu. "Terrain is all about making the tools for this kind of rapid iteration possible for delivering better RNA."