Meet our Amazing Clients

• Pioneering leader in genetic medicines, with proprietary platform based on Nobel Prize-winning technology: 4DMT is applying the principles of directed evolution to invent novel, customized AAV vectors to combat the shortcomings of conventional vectors. With the strategic appointment of three industry veterans and the formation of an Ophthalmology Advisory Board, 4DMT is rapidly advancing its commercialization efforts as it prepares for pivotal Phase 3 trials in 2025.
• Quality synthetic vectors fit for purpose: Clinical data supports the therapeutic potential of 4DMT’s innovative approach to genetic medicines, including 4D-150, the company’s lead ophthalmology candidate which has the potential for multi-year to lifelong durability in wet AMD following a single routine injection
  • Each 4DMT candidate has been designed with optimized capsids that are delivered by the routine route of administration for each disease – including intravitreal for eye diseases, aerosol for cystic fibrosis (CF), and intravenously for Fabry disease.
• Key Events in 2025: Initiation of global 4FRONT Phase 3 clinical trial of 4D-150 in wet AMD in Q1 2025; 52-week landmark analyses for both severe disease activity (Dose Expansion) & broad wet AMD disease activity (Population Extension) cohorts of PRISM Phase 1/2 clinical trial of 4D-150 in February 2025; Initiation of Phase 3 clinical trial of 4D-710 in CF lung disease in H2 2025

• Potential Functional Cure for Hepatitis B (HBV): HBV is still seen as a major public health crisis with more than 290 million chronic carriers globally. Chronic hepatitis B (CHB) is the primary cause of liver death worldwide and mortality rates continue to rise. Aligos is developing ALG-000184, the first novel, oral drug candidate for the treatment of HBV infection that can inhibit multiple components of the viral lifecycle, leading to more complete suppression of the virus compared to other current standards of care.
• MASH is Hot Again: Following the approval of Madrigal’s Rezdiffra, the MASH space is heating up again with additional companies getting closer to the FDA finish line. Aligos is developing ALG-055009 as a potentially best-in-class THR-ß agonist, offering 5-50x greater potency than competitors with a focus on enhanced beta selectivity and optimized pharmacokinetics. Recent Phase 2a HERALD study data supports its superior safety profile with fewer side effects, positioning ALG-055009 as a frontrunner in MASH treatment and significantly improving patient outcomes.
• Key Events in 2025: Data presentations on ALG-055009 & ALG-000184 at AASLD’s The Liver Meeting in November 2024; IND submission & initiation of a Phase 2 study of ALG-000184 for CHB expected in Q1-2025; Completion of activities required for a Phase 2b study of ALG-055009 in MASH expected by the middle of 2025

• A new alternative to opioids, offering ultra-sustained, controlled and targeted pain relief without the high risk of addiction: Current post-operative pain management only offers short-term relief, necessitating frequent re-dosing of systemic analgesics like opioids, which can lead to dependency and side effects, especially during extended recovery periods. Allay’s pipeline programs use biodegradable polymer-based platforms to provide sustained, localized pain relief at the target site for weeks. This approach reduces reliance on opioid re-dosing, minimizes the need for clinical oversight, and supports a smoother transition into rehabilitation, offering a more effective and safer alternative for long-term post-surgical pain management.
• Revolutionizing pain management after total knee replacement: Allay’s lead product candidate, ATX101, is a sustained-release therapy designed to provide extended pain relief following total knee replacement (TKR) surgery with a single application, aiming to reduce the need for repeated dosing and systemic painkillers. Recent data from their dose-ranging Phase 2B trial demonstrated up to four weeks of sustained post-surgical pain relief, significantly reducing opioid use and improving recovery and daily functioning compared to standard care. The FDA’s recent IND acceptance of ATX101 for a Phase 2B trial further supports its breakthrough potential and positions ATX101 as a frontrunner to transform post-surgical pain management for TKR patients.
• Key Events in 2025: Initiation of Phase 2B registration trial of ATX101; Advancement of other post-surgical pain products for additional indications.

• Class-Leading Lean Mass Preservation Data: Pemvidutide, Altimmune’s GLP-1/Glucagon dual receptor for obesity and MASH has demonstrated lean mass preservation superior to what’s been historically reported with diet and exercise, with a lean loss ratio of only 21.9%, and visceral adipose tissue, a risk factor for cardiovascular disease, reduced by 28.3%.
• 1:1 Balanced GLP-1 + Glucagon Agonism is a Key Differentiator from Similar MOAs: The glucagon activity in pemvidutide has a direct effect on the liver and is intended to reduce cholesterol, triglycerides, LDL, and liver fat in a more profound way than other incretin-based approaches that also incorporate glucagon but favor GLP-1.
• Breakthroughs in Obesity & MASH Clinical Trials: At 48 weeks of treatment in the MOMENTUM trial for obesity, subjects receiving pemvidutide achieved weight loss of up to 15.6% with weight loss continuing at the end of treatment. Additionally, in the IMPACT trial for MASH, subjects achieved up to 68.5% relative reduction in liver fat content, an important predictor of MASH resolution and fibrosis improvement.
• Key Events in 2025: Initiation of pivotal Phase 3 obesity program which includes four clinical trials evaluating pemvidutide in a variety of patient populations with obesity, Q2 2025 Phase 2b readout in MASH (IMPACT Trial), Pipeline expansion – 1H 2025 initiation of at least one mid-stage trial evaluating an additional pemvidutide indication.

• Harnessing computational chemistry, AI and quantum molecular dynamics to design dual-binding small molecules: Traditional therapies often target single disease components, limiting their effectiveness against complex conditions. Automera’s innovative autophagy-targeting chimeric molecules (AUTAC) platform generates novel therapies that degrade not only disease-related proteins but also protein aggregates, malfunctioning mitochondria, and pathogens, eliminating a range of disease-causing agents. AUTACs have the potential to provide a more flexible approach to protein degradation than existing approaches such as proteolysis-targeting chimeras (PROTACs) to trigger protein degradation.
• Accelerating R&D with Singapore’s biotech ecosystem: Automera’s R&D is based in Singapore, leveraging the country’s efficient regulatory system, advanced biotech infrastructure, and access to diverse patient populations. This strategic location accelerates clinical trials, while Singapore’s multicultural demographic ensures inclusive recruitment, providing broad, globally relevant data that strengthens the development of Automera’s therapies for diverse patient groups.
• Key Events in 2025: With an initial focus on cancer, continue advancing early pipeline assets and refining its platform.

• Jean Cui, Ph.D., Founder, Inventor, and Drug Designer: Dr. J. Jean Cui, Scientific Founder, President and CEO of BlossomHill Therapeutics, has become a household name due to her previous successes. Her BlossomHill team is building brand new cancer therapies from the ground up to address existing treatment limitations, including resistance, rather than building on existing therapies or science.
• A Pandemic Idea Come to Fruition: BlossomHill’s pipeline was spawned while Jean was confined to her living room during the pandemic. The company flew under the radar when repotrectinib was approved last fall – one of three drugs invented by Jean now on the market. After securing $100M in a Series B financing, the company is advancing its oncology programs and continuing to expand its pipeline of oncology and autoimmune discovery programs.
• Key Events in 2025: Progressing Phase 1 clinical trial investigating BH-30236-01, a multi-targeted CLK inhibitor, in relapsed or refractory adult acute myeloid leukemia (AML) and higher-risk myelodysplastic syndrome (HR-MDS).

Advancing next generation approaches for diseases with unmet need

• Unmet need in obesity treatment still exists: Despite limitations and side effects, weight loss drugs represent more than $6 billion in annual revenues globally, with projections reaching $150 billion by 2030 (Reuters). CinRx’s CinCo, CinFina, is advancing new approaches to treating obesity and preserving lean muscle mass.
• Validating data across lead obesity programs: Recently presented at Obesity Week in November, CinFina’s CIN-110’s ability to safely and tolerably reduce caloric intake while quickly decreasing body weight after just a single dose positions it as a target=”_blank” high-potential treatment candidate. CIN-109 also produced meaningful weight loss and was well-tolerated across all dose levels in Phase 1 multiple ascending dose study, including at the highest dose tested, where most weight loss was due to a reduction in fat mass while increasing lean mass.
• The CinRx Difference—a successful “hub and spoke” model: CinRx’s portfolio of CinCos are centrally funded and managed by a team of scientific, technical, and drug development experts with decades of experience. Among the CinCos, are CinFina, CinDome and CinPhloro.
• Key Events in 2025: Additional data expected for CinFina’s CIN-110 Phase 1 single ascending dose (SAD) study; CinPhloro’s ongoing Phase 2 enviva study of CIN-103 for IBS-D evaluating the safety, efficacy and tolerability.

• Matching the Right Patient to the Right Treatment with Machine Learning and CRISPR Gene-Editing Technology: Engine’s NetMAPPR platform combines machine learning and CRISPR gene-editing technology to uncover key biomarkers that match patients with the most effective targeted treatments. By decoding complex genetic networks, they’ve identified over 30 new precision medicine opportunities, driving innovative therapies from concept to clinic and opening doors for strategic cancer research partnerships.
• Building a Robust Pipeline of Precision Medicines across Multiple Diseases: Engine is rapidly expanding its drug development pipeline with over 15 biomarker programs in clinical stages. By identifying novel drug targets and combining therapies across multiple gene pathways, Engine is advancing a diverse pipeline of next-generation treatments that promise to address unmet medical needs in oncology, neurology, and beyond.

• Overcoming Drug Resistance with Dual-Payload ADCs: Hummingbird Bioscience is pioneering a unique strategy against drug resistance in cancer therapy by engineering multiple payloads into the same ADC. Their first dual-payload ADC, HMBD-802, combines a TOP1 inhibitor, exatecan, with an undisclosed DNA damage response inhibitor. This combination, carefully balanced with a 4:4 potency ratio, has shown promising preclinical efficacy in overcoming drug resistance. They’re also advancing traditional monoclonal antibodies like HER3-targeting HMBD-001 and VISTA-targeting HMBD-002 in clinical trials.
• Versatile Platform for Multiple Therapeutic Modalities: Hummingbird Bioscience’s Rational Antibody Discovery (RAD) platform is uniquely designed to engineer highly selective antibodies for a wide range of cancer therapies, including ADCs, bispecific T-cell engagers (TCEs) and monoclonal antibodies. By using AI-powered insights and immunoengineering, the platform works to overcome challenges like tumor resistance and optimize the targeting of hard-to-drug proteins.

• The first in vivo gene editing therapeutic to be tested in infants: Following clinical trial clearances in the US, UK and Australia, iECURE’s ECUR-506 is poised to be the first in vivo gene editing candidate to be tested in infants in the US.
• A potentially curative therapy for a deadly disease that has long stymied therapeutic developers: For decades, genetic medicine pioneers have pursued gene therapy approaches to treat ornithine transcarbamylase (OTC) deficiency to no avail, ECUR-506 may finally solve this challenge.
• Key events in 2025: Interim data readouts on safety and indicators of efficacy for ECUR-506 in neonatal onset OTC deficiency, potential advancement of other pipeline programs.

• Democratizing CAR-T Therapy: Kelonia’s best-in-class in vivo gene placement system (iGPS) enables the efficient delivery of genetic medicine with high tissue specificity and avoids costly and lengthy ex vivo manufacturing, burdensome lymphodepleting chemotherapy and reliance on large research hospitals
• $800M Collaboration with Astellas aims to develop universal in vivo CAR-T therapies and has potential applications in cancer and autoimmune conditions
• Key Events in 2025: IND for lead candidate KLN-1010 in multiple myeloma, Astellas partnership progression

• Menin Inhibitor Race: Among competitors, Kura’s ziftomenib stands to be best-in-class. The latest encouraging data on ziftomenib as a combination therapy with standard of care therapy, cytarabine plus daunorubicin (7+3), in NPM1-mutant and KMT2A rearranged AML included a 100% CR rate. Ziftomenib also received FDA Breakthrough Therapy Designation as a monotherapy in relapsed/refractory NPM1-mutant AML. Kura is set to present additional Phase 1a combination data from KOMET-007 by the end of the year.
• $1.2B Vote of Confidence: In November, Kura entered into a $1.2 billion partnership with Kyowa Kirin to develop and commercialize Ziftomenib outside of the US. Kura received $330 million upfront and expects to receive up to $420 million in near-term milestone payments, including a payment upon the launch of ziftomenib in the monotherapy relapsed/refractory setting. In addition, Kura is eligible to receive additional development, regulatory and commercial milestone payments of $741 million, totaling up to $1.161 billion in payments for milestones and the opt-in for solid tumor indications.
• Specialists in the long game: Kura continues to progress with stability and rigor. In 2024 they announced a $150M private placement, fueling the company as they continue potentially practice-changing research.
• Key Events in 2025:
  • Pivotal topline data from KOMET-001 of ziftomenib in relapsed/refractory NPM1-mutant AML
  • Continued expansion of KOMET-007 Phase 1b trial with ziftomenib in dose escalation combination with 7+3 and venetoclax and azacitidine (ven/aza)
  • Initiation of proof-of-concept study for ziftomenib in gastrointestinal stromal tumors (GIST), the first target of solid tumors for ziftomenib
  • Nomination of next generation menin inhibitor; research will be conducted in additional oncology targets as well as diabetes
  • Present data from KURRENT-HN trial of tipifarnib and alpelisib in PIK3CA-dependent head and neck squamous cell carcinoma (HNSCC)

• Mastering Mucosal Immunity: Netherlands and Singapore-based biotech, Leyden Labs, specializes in targeted antibody delivery via nasal sprays to stop viral infections at the point of entry – the nasal mucosa.
• Universal Defense Against Respiratory Viruses: Focused on broad and enduring protection against evolving respiratory viruses, Leyden Labs is developing novel therapies such as, PanFlu™, which targets conserved viral regions to ensure long-term effectiveness against new strains. By harnessing this approach, Leyden Labs aims to create self-administered, accessible treatments that provide patients with immediate autonomous protection against both current and future viral threats.
• Expert Insights from Leyden Labs’ CEO: Leyden Labs CEO, Koenraad Wiedhaup, can speak on a wide range of topics, including: the future of vaccines for broader infectious diseases, the evolving biotech landscape in Europe, preparing for the next global pandemic, leading international biotech operations, navigating the complexities of biotech financing and more.

• Growth and Expansion in CAR-T: In October, Lyell announced the acquisition of ImmPACT Bio to expand its focus on next generation CAR-T development. This acquisition strengthened Lyell’s pipeline with a dual-targeting CD19/CD20 CAR T-cell product candidate, IMPT-314, in hematological malignancies. This is paired with Lyell’s existing differentiated CAR T-cell product candidate, LYL-119, in solid tumors. Lyell is committed to advancing all types of CAR T-cell therapies in cancer types with gaps in treatment.
• Supported by the Data: Lyell ended 2024 with new Phase 1/2 data from the IMPT-314 in B-cell Lymphoma at the American Society of Hematology (ASH) annual meeting. Data show a 100% objective response rate within 28 days and a 73% complete response rate within three months of treatment.
• Key Events in 2025:
  • Progression of LYL-119 and IMPT-314 programs in solid tumors and hematological malignancies respectively.
    • LYL-119: Initial clinical data anticipated from Phase 1 trial in patients with ROR1‑positive solid tumors
    • IMPT-314: Initiate pivotal trial in third line relapsed/refractory large B-cell lymphoma in the third-line setting

• Maintaining Optimal Levels of PTH: With Phase 2 trial design and rationale established, MBX-2109 has demonstrated ability in Phase 1 for once-weekly dosing to deliver consistent and optimal parathyroid hormone (PTH) levels. This offers significant promise for patients with hypoparathyroidism (HP) who must account for low PTH by constantly taking supplements to maintain blood calcium levels
• Platform for Precision: Based on technology from the lab of peptide pioneer Richard DiMarchi, Ph.D., MBX is overcoming a key limitation of peptides as medicine – short half-life that mandates repeat dosing and variability in blood concentrations – with treatments designed to have sustained effect in patients for better convenience and outcomes
• The Obesity Race: MBX 4291 is a long-acting GLP-1/GIP Receptor co-agonist, in development as a potential treatment for obesity. This preclinical therapy has the potential for improved GI tolerability, less frequent administration and reduced dosing complexity.
• Key Events in 2025: Following the closing of an upsized IPO in September of 2024, MBX has key upcoming milestones in 2025 including topline study results for the Phase 2 MBX-2109 trial in patients with HP expected in Q3 2025. MBX is also advancing a second program, MBX-1416, for potential treatment of post-bariatric hypoglycemia in its Phase 1/2 clinical trial

• Helping ADCs Find Their Fate: Mythic is pioneering self-navigating ADCs. Its approach manipulates the trafficking of antibody drug conjugates in cells with its FateControl platform to increase uptake in tumors while avoiding payload release in healthy tissues – potentially increasing therapeutic potency and improving tolerability.

• Well-Known Target, Common in NSCLC? It’s KisMET: Therapeutic pipeline is led by KisMET-01 clinical study of MYTX-011, a cMET-targeting ADC, for treatment of NSCLC where 70% of tumors have cMET over-expression. Mythic’s approach allows its ADCs to target cMET in patients with low- to – moderate target expression, a potential game changer as currently available ADCs require high target expression to be effective.

• Key Events in 2025: Data from KisMET-01 trial in NSCLC

• R&D Engine Powering Growth following the $1.2 billion deal with Sanofi to Co-commercialize its COVID-19 Vaccine globally and the potential of multiple revenue streams. We are focused on leveraging our proven technology platform by developing early- and late-stage assets through research and development (R&D) and partnering on these assets, as well as our Matrix-M™ adjuvant, with the goal of protecting public health.
• Differentiated Technology Platform: Novavax offers a differentiated vaccine platform that combines its proprietary Matrix-M adjuvant and innovative recombinant, protein-nanoparticle to enhance immune response. Matrix-M uses saponins, naturally occurring compounds in the bark of the Soapbark tree, to help induce a stronger, more potent and longer-lasting immune response. This technology is used in Novavax-developed vaccines and can be licensed out for use in a wider array of vaccines. The nanoparticle helps the immune system recognize a virus from different angles and can provide a greater dose of antigen in a safe and targeted manner.
• Key Events in 2025:
  • Continued Progress of Sanofi partnership
  • Progress pivotal Phase 3 clinical trials of seasonal influenza and COVID-19-Influenza Combination (CIC) vaccines and continue to work with FDA to assess potential path for accelerated approval
  • Progress strategy to identify potential partners and out-licensing opportunities for our technology platform
  • Further advance early-stage pipeline, starting with our core of infectious disease and beyond

• A leader in innovative, pathway-specific genetic medicines: Genetic cardiomyopathies, caused by mutations in over 30 genes linked to cardiac force transmission, represent a major unmet medical need, particularly since most mutations are inherited in an autosomal dominant pattern, making traditional gene replacement therapies unfeasible. Nuevocor’s mechanobiology PrOSIA platform enables the development of targeted genetic medicines by identifying shared biomechanical pathways in genetic cardiomyopathies. This approach surpasses traditional gene replacement therapies, offering a comprehensive treatment strategy.
• Targeting the Root Cause of Lamin-Related Dilated Cardiomyopathy: There are no effective treatments beyond heart transplant for Lamin-related dilated cardiomyopathy (DCM). Nuevocor’s lead program, NVC-001, targets the biomechanical root cause of this aggressive disease. Data presented at ESGCT and AHA demonstrated promising therapeutic benefits and a strong safety profile in preclinical models, showing the potential for NVC-001.
• Key Events in 2025: IND submission and initiation of clinical trials for NVC-001; Potential advancement of other pipeline programs

• Unlocking the Secrets of Bats: With over 65 million years of evolutionary resilience, bats have evolved extraordinary adaptations that allow them to thrive in extreme environments, reduce inflammation, tolerate viruses, and exhibit impressive longevity. With 95-97% genomic similarity to humans, Paratus discovered that bats provide an untapped model for studying the biological mechanisms of human disease – holding unique evolutionary traits that offer insights into key health challenges related to inflammation, metabolism, and immune function.
• Integrated Discovery Platform: Leveraging one of the largest collections of bat genomes and tissue samples, Paratus’ platform combines cutting-edge genomics and bioinformatics to rapidly identify novel therapeutic targets for challenging disease areas with large unmet need.
• Leveraging Global Expertise: Paratus brings together the brightest minds from both Singapore and the US to translate bat biology findings into drug discovery and development. By tapping into the expertise of leading researchers, clinicians, and industry leaders across two continents, Paratus fosters a dynamic, cross-border team that accelerates the development of next-generation therapies.

• Two Approaches in the Clinic for IL-2: Lead clinical programs utilize a familiar cytokine (IL-2) in new ways, including as a highly selective partial agonist designed to avoid toxicities and in an orthogonal system designed to control activity, durability and persistence of a CD19 CAR-T
  • Unveiled positive initial clinical first-in-human data from Phase 1a/1b clinical trial of its α/β biased IL-2 partial agonist, STK-012 in solid tumors at AACR 2024
• Dynamic pipeline driven by cytokine engineering platforms across oncology, autoimmune & inflammatory indications:
  • Three platforms – partial agonists, orthogonal systems and surrogate cytokine agonists
  • Advancing CAR-T therapy for autoimmune into new clinical areas, including systemic lupus erythematosus (SLE)
• Key Events in 2025: Advancing lead clinical programs through clinical investigation to initial clinical trial data readouts; Advancing additional partial agonist systems toward clinical investigation; building on partnerships with Sanofi (2024) and Merck (2022), Expanding orthogonal system for autoimmune disease into SLE without use of lymphodepletion and other disease areas

• Pioneers in novel aging mechanisms: Through their senolytic platform, UNITY is developing a novel therapeutic approach to remodel the retina. They are the first company to produce Phase 2 proof-of-concept data that validates their senolytic approach by targeting destructive senescent cells in retinal tissues, while preserving healthy cells, a potentially paradigm shifting approach to treating vision loss in diabetic macular edema (DME).

• Sights set on age-related vision loss: Lead senolytic candidate UBX1325 has produced impressive data as an entirely novel mechanism of action showing that one injection not only stabilized retinal structure but improved vision for almost one year in a very difficult-to-treat DME patient population – a clear benefit over current medicines that require frequent injections for diminishing benefits. Now, the company is conducting a Phase 2b study to discern how their senolytic therapeutic approach compares head-to-head with current standards of care.
• Key Events in 2025:Initial head-to-head Phase 2b ASPIRE data in DME anticipated in Q1 2025; and 36-week data expected in 2Q 2025.

• Antibodies Engineered with Purpose: Xencor’s Fc domain technology supercharges antibody therapies by extending half-life, enhancing immune activation, and enabling precise targeting. It powers a new wave of biologics designed for superior efficacy against complex diseases.
• I&I is Red Hot. How do Bispecific Antibodies play into it? Xencor’s plug-and-play antibody approach allows for the extension into multiple disease areas beyond oncology, including autoimmune and inflammatory diseases, as evidenced by multiple newly announced T-cell engager programs with upcoming clinical milestones.

• Validated Commercial Platform: Xencor’s protein engineering platform is validated through multiple commercially approved drugs and continues to be a highly sought after partner for novel drug development (over 10+ select partnered/licensed programs and 8 owned programs in various disease areas).

• Key Events in 1H 2025: Phase 1/2 Data and Study Initiations Across Immunology and Oncology Programs
  • Immunology Portfolio
    • XmAb942: SAD readout in inflammatory bowel diseases
    • Plamotamab: Initiation of Phase 1/2 study in rheumatoid arthritis
  • Oncology Portfolio
    • XmAb819 and XmAb808: Initiation of dose expansion in ccRCC and solid tumors.
    • Vudalimab: Mono and combo cohort expansion readout in mCRPC. Evaluate chemo combination safety in NSCLC.

• Dual Pathway Approach to Improve Patient Outcomes: The autoimmune space is plagued with clinical failures of biologics targeting single pathways in complex diseases driven by multiple pathways. Zura is developing a pipeline of novel dual-pathway antibodies addressing validated disease drivers that have potential to improve outcomes and restore hope for those burdened by autoimmune diseases.
• First-in-Class Treatment for Systemic Sclerosis (SSc): SSc, a rare and life-threatening autoimmune disease characterized by tissue inflammation and fibrosis, has been a challenging indication for drug development. Zura is advancing tibulizumab, a “four-headed hammer” dual agonist of IL-17 and BAFF to address both pathways which are known drivers of SSc. A potentially first-in-class treatment for SSc, tibulizumab is a fusion of FDA approved TALZ® and clinically validated tabalumab, with potential for broader efficacy and a more convenient dosing regimen than existing approaches.
• Key Events in 2025: Initiation of Phase 2 study of tibulizumab in SSc in Q4-2024; Initial indication selection for a Phase 2 clinical study of crebankitug expected by the end of 2024; Initiation of Phase 2 study of tibulizumab in HS in Q2-2025