▎WuXiEdited by Kant Content Team

Today, a well-known media website in the biopharmaceutical industryBioSpaceSelected as2024Annual New Generation of Biotech Rising Stars (NextGen Class of 2024）。Through a comprehensive evaluation of the company's financing situation, collaborations, R&D pipeline, growth potential, and innovation,BioSpaceSelect the rising stars in life sciences that made the list. Those on the list this year30Jiaxin fully demonstrates the impact of new treatment models and innovative drug development strategies on new drug development., with a wide range of focus areas, includingGene Therapy, Cell Therapy,RNATherapy、RadiopharmaceuticalsAnd innovative small moleculesetc.DifferentModel. The WuXi AppTec content team, based on this report and combined with publicly available information, introduces these shining stars in the field of bioscience to our readers.

Notably, among these listed supernova companies,Nine leaders of the company will also attend.The 12th Session Held on January 9, 2024WuXi AppTec Global Forum, bringing unique insights from diverse perspectives as well as new prospects for novel molecular therapies. Professionals in the global pharmaceutical and healthcare fields are welcome to attend.

▲The 12th WuXi AppTec Global Forum will be held in San Francisco on January 9, 2024, from 1:30 PM to 6:00 PM (PST). Scan the QR code or click "Read More" at the end of the article to apply for attendance.As with previous editions, the 2024 WuXi AppTec Global Forum operates on an "invitation-approval basis" and does not charge any participation fees. Professionals in the global pharmaceutical and healthcare fields are welcome to register.

1. Rapport Therapeutics

Rapport Therapeutics' precision medicine development strategy utilizes receptor-associated proteins to target genetically and clinically validated targets.Due to the more specific distribution of receptor-associated proteins, this strategy allows for targeted receptors in specific brain neuroanatomical regions that trigger neurological diseases. It can not only improve the efficacy and tolerability of existing therapies but also provide new strategies for neurological disorders that currently lack effective treatments.Rapport Therapeutics announced the completion of a $100 million funding round in March last year.Series A Financing, itsChief Executive Officer (CEO) Abraham Ceesay Will Attend in 2024WuXi AppTec Global Forum, share insights on the next wave of innovative therapy development.

2. Apogee Therapeutics

Apogee Therapeutics is a biotechnology company dedicated to advancing novel, potentially "best-in-class" therapies to meet the needs of millions of patients with immune and inflammatory diseases. The company was founded in 2022 by Fairmount and Venrock Healthcare Capital Partners and went public last year.2In August 2023, Apogee Therapeutics' lead candidate drug APG777 completed the first patient dosing in a Phase 1 trial. The drug is being developed for the treatment of atopic dermatitis and other inflammatory diseases.APG777 is also in the preclinical development stage for asthma. The company completed a $149 million financing in December of last year.Series B Financing。

3. CARGO Therapeutics

CARGO Therapeutics is dedicated to developing the next generation of CAR-T cancer therapies.Its potential "first-in-class" and "best-in-class" CD22-targeted CAR-T therapy, CRG-022, achieved sustained remission in over 50% of large B-cell lymphoma (LBCL) patients who were already resistant to CD19-targeted CAR-T therapy in a Phase 1 clinical trial.Based on this result, the FDA has granted it Breakthrough Therapy designation, and currently, the therapy has entered Phase 2 clinical stage.

CARGO Therapeutics completed a $200 million financing in March last year.Series A Financing，Crystal Mackall, M.D., Co-Founder, Founding Director of the Stanford Cancer Cell Therapy Center, and Pioneer in T-cell Research, to Attend 2024.WuXi AppTec Global Forum, Participate in panel discussions.

4. Aera Therapeutics

Aera Therapeutics, founded by gene-editing pioneer Professor Zhang Feng, aims to utilize a human protein named PEG10, which can self-assemble into structures resembling viral capsids, for the delivery of RNA, DNA, and gene-editing systems. Last year, the company completed $193 million in Series A and B funding rounds.This vector has lower immunogenicity compared to viral vectors and is expected to reduce the toxic side effects of gene therapy.

5. Nido Biosciences

Nido Biosciences is dedicated to leveraging a deep understanding of human genetics and diseases to develop innovative small-molecule therapies for neurological disorders.Its lead investigational therapy, NIDO-361, has entered the clinical development stage for the treatment of patients with Spinal and Bulbar Muscular Atrophy (SBMA).NIDO-361 is an innovative small molecule that binds to a unique site on the androgen receptor and corrects transcriptional dysregulation to restore healthy cellular function. The company achieved this in May of last year.AnnouncementCompleted seed round, Series A, and Series B financing, with a total amount reaching $109 million.

6. ReNAgade Therapeutics

ReNAgade Therapeutics is an RNA therapy research and development company distinguished by its delivery platform and proprietary tools. This platform integrates its proprietary delivery technologies, including novel lipid nanoparticles, with a broad range of RNA-based coding, editing, and gene insertion tools.ReNAgade Therapeutics aims to address the major limitations of RNA therapies by enabling the delivery of RNA drugs to previously inaccessible tissues and cells in the body, thereby expanding the range of potentially treatable diseases.Last May, ReNAgade announced the completion of a $300 millionSeries A FinancingThe funds are expected to be used to advance the further development of preclinical and clinical pipelines for RNA drugs.

7. Orbital Therapeutics

Orbital Therapeutics is an RNA therapy company formed by Beam Therapeutics, a star company in gene editing.Orbital aims to gather and integrate existing and emerging RNA technologies and delivery mechanisms to build a unique RNA technology platform. The goal of this platform is to extend the durability and half-life of innovative RNA therapies and deliver them to a variety of different cell and tissue types.Based on this platform, the company's R&D pipeline will include vaccines, immunomodulatory drugs, protein replacement therapies, and more.

Last April, Orbital completed a $270 millionSeries A Financing。Giuseppe "Pino" Ciaramella, Ph.D., Co-founder, CEO and President of the company, will also attend 2024.WuXi AppTec Global Forum, jointly discussing how emerging treatment models are leading the industry into a new round of transformation and breakthrough.

8. Abdera Therapeutics

Abdera Therapeutics focuses on developing technologies that utilize targeted antibodies to deliver radioactive elements to tumor targets.Novel Radiopharmaceuticals,This class of drugsCapable of modulating its pharmacokinetics to overcome the metabolic limitations of conventional small molecules or large protein molecules, thereby enhancing therapeutic efficacy.Its DLL3-targeted small cell lung cancer therapy is planned for IND submission in 2024. Ms. Lori Lyons-Williams, the company's president and CEO, mentioned at the third event of the WuXi AppTec "BOLD: Innovation Leads, Brave the Future" series: "The power of radiopharmaceuticals lies in their highly potent and targeted payload."We believe that in the next 10 years or so, radiopharmaceuticals will truly transform the way cancer patients are treated.”

9. SonoThera

SonoThera's technology platform is based on a biophysical process known as sonoporation. This principle involves the release of energy when microbubbles are burst by ultrasound, creating small pores in cell membranes to facilitate the delivery of gene therapy. These pores in the cell membrane are only temporarily present and will close automatically.This technology can be used to facilitate the delivery of gene therapies of various types and sizes without the need for viral vectors.The delivery of contrast agents mediated by microbubbles has been widely used in clinical practice and demonstrated good safety.The company announced in December 2022 the completion of a $60.75 millionSeries A Financing`, the funding is expected to be used for further development of the company's ultrasound-guided non-viral gene therapy platform.`

10. Bitterroot Bio

Bitterroot Bio focuses on harnessing the power of immune modulation to treat cardiovascular diseases, leveraging the latest advances in immunology to discover new targets and develop innovative protein therapies.The company's lead project, BRB-002, targets the CD47/SIRPα pathway, aiming to address the underlying dysfunction that leads to atherosclerosis.

According to the industry media Fierce Biotech, BRB-002 differs from similar drugs used in tumor treatment in several key aspects. First, its formulation reduces the likelihood of adverse reactions that occur with other CD47-targeting drugs. Second, it has a higher degree of purification, which increases its affinity for the CD47 target, thus requiring a lower dosage. Moreover, it only needs to enter the blood vessel walls without penetrating the tumor, so a small amount of the drug can show effects in cases of atherosclerosis. The company completed$145 millionSeries A Financing。Bitterroot Bio's CEO and President, Pavan Cheruvu, M.D., to Attend 2024WuXi AppTec Global Forum, Discuss the latest R&D and breakthrough trends in the industry.

11. Belharra Therapeutics

Belharra Therapeutics hopes to expand the targeting range of small-molecule drugs by significantly increasing the identification of protein sites that can bind with ligands.The team uses a library of probes to comb the surface of proteins, searching for undiscovered pockets. The strategy adopted by Belharra is to directly analyze proteins within cells, rather than studying only purified proteins or static snapshots from structural research. This approach allows Belharra to gain a more accurate understanding of protein interactions in the complex environment inside cells, thereby increasing the likelihood of developing effective drugs.

In addition, Belharra scans all protein surfaces they discover using a library of non-covalent molecular probes. This technique enables scientists to study sites that cannot be identified through typical covalent labeling methods with greater precision, thereby enhancing the identification of potential protein-ligand binding pairs.Belharra Therapeutics has identified over 4,000 new small molecule-protein pairs, including those involving floppy, tricky, and pocket-hidden proteins.The company in January last yearCompleted a $50 millionSeries A FinancingAnd step out of stealth mode.

12. Kate Therapeutics

Kate Therapeutics’ DELIVER platform combines diverse capsid library generation with stringent transcription-based in vivo selection and machine learning to develop functional capsid variants with the potential to target any cell of interest in living models.Sharif Tabebordbar, Ph.D., Co-founder and Chief Scientific Officer of Kate Therapeutics, generates millions of different viral variants, which are then injected into mice or monkeys. By sequencing animal tissues, he can determine which viral variant is more effective at transfecting muscle.Based on its research, KATE produced a capsid named MyoAAV.

Astellas Pharma Signs Agreement with Kate Therapeutics for Exclusive License to Develop, Manufacture, and Commercialize KT430, a Candidate Drug Aimed at Treating X-Linked Myotubular Myopathy.Kevin Forrest, Ph.D., Co-Founder, CEO and President of Kate Therapeutics, was also invited to participate in 2024.WuXi AppTec Global Forum, discussing new trends in the biopharmaceutical frontier with thought leaders from different fields of the industry.

13. Convergent Therapeutics

Convergent Therapeutics' lead investigational therapy, CONV01-α, is a monoclonal antibody conjugated with Actinium-225 (Ac-225), targeting prostate-specific membrane antigen (PSMA). Actinium-225 is a radioactive alpha particle emitter. A key functional characteristic of CONV01-α is that once it binds to PSMA, it becomes internalized, delivering its potent radioactive payload directly into prostate cancer cells while minimizing damage to surrounding healthy tissue.CONV01-α is expected to become the first Ac-225 radiolabeled antibody approved for the treatment of prostate cancer.The company completed a $90 million Series A financing round in May of last year.

Philip Kantoff, Ph.D., Co-founder and CEO of the company, stated during the recently concluded "BOLD: Innovation Leads, Courageously Embracing the Future" Series Episode 4 event,Radiopharmaceuticals are not a new approach, but rather an improvement in the delivery method of radiation. This allows radiation to be delivered precisely in liquid form to the location of the tumor, minimizing toxicity to non-target areas while maximizing the concentration of targeted radiation.This advancement has greatly propelled the development in this field.Dr. Kantoff will also attend in 2024.WuXi AppTec Global Forum, sharing its profound insights in cancer treatment.

14. Cajal Neuroscience

Cajal Neuroscience is a company focused on studying the mechanisms, spatial and temporal complexity of neurodegenerative diseases. By combining its expertise in neuroscience, neuroanatomy, and computational biology with cutting-edge technology, Cajal discovers how and when hundreds of genetic signals and numerous pathways lead to idiopathic neurodegeneration and related disease symptoms.

Cajal's industrialized light-sheet microscopy platform enables researchers to directly observe the biological effects of specific targets or therapies in an intact brain. Through 3D imaging, light-sheet microscopy can analyze complex spatial phenotypes in the brain, such as the spread of pathological changes and selectively vulnerable regions.The company completed a $96 million financing at the end of November last year.Series A Financing。

15. Matchpoint Therapeutics

Matchpoint TherapeuticsMatchpoint Therapeutics aims to develop innovative covalent small-molecule drugs, with its R&D pipeline initially focusing on the field of immunology. The company's goal is to target points that have been validated by human genetics or clinical studies, developing therapeutic approaches utilizing covalent chemistry.The company's Advanced Covalent Exploration (ACE) platform integrates various proprietary tools and has the potential to revolutionize covalent drug discovery and development., including a chemical proteomics platform to discover innovative covalent binders that target disease-related proteins. This platform can screen low-abundance targets within their native cellular environment.

In addition, the platform's machine learning algorithms can uncover principles of covalent drug discovery, guide target prioritization, and predict compound scaffolds, supporting medicinal chemistry and compound library design. It also continuously optimizes and expands its proprietary covalent compound library based on predictive algorithms. The company came out of stealth mode in October 2022.And announced that it has secured $100 millionFinancing。

16. Bonum Therapeutics

Bonum focuses on developing protein therapies with controllable activity. Its main research directions currently include cytokines with controllable activity (such as IL-12, IFNα, and TGFβ), as well as anticancer therapies activated under specific conditions.The therapeutic molecules designed by the company itself carry binding domains that can act as sensors. When these domains bind to their targets, they activate the activity of the therapeutic molecules. Their sensor protein domains can bind to peptides, proteins, or metabolites. In addition to cancer treatment, this technology has exciting application potential in treating autoimmune diseases, metabolic disorders, and pain relief.The company completed a $93 million financing in November 2022.Series A Financing。

17. Juvena Therapeutics

Juvena Therapeutics' platform combines databases revealing the connection between secreted proteins and specific diseases, computer screening technology, experimental screening using human cells and animal models, and protein engineering capabilities, creating a highly integrated approach to accelerate the development of biologics for chronic and age-related diseases.

The company was co-founded by Dr. Hanadie Yousef, an expert in the biology of aging and degenerative diseases, and Dr. Jeremy O'Connell, an expert in systems biology and proteomics. In November 2022, it completed a $41 millionSeries A Financing, expand the company's drug discovery platform, and advance the company's biologics pipeline targeting chronic and age-related diseases.

18. Georgiamune

Georgiamune Focuses on Reprogramming Immune Signaling Pathways to Redirect the Immune System Against DiseaseThe company was founded by Dr. Samir N. Khleif based on his discoveries in new mechanisms for modulating immune responses. Dr. Khleif's research has identified novel core targets that control key immune cell pathways, leading to the development of molecules that can modulate and restore these immune signaling pathways for the treatment of cancer and autoimmune diseases.

Georgiamune has a total of 8 ongoing research projects, with its leading project, GIM-122, being a bifunctional monoclonal antibody. GIM-122 is designed to overcome immunotherapy resistance in cancer patients and is intended for advanced malignant solid tumors that have failed immune checkpoint inhibitor treatments. The IND application for this therapy received FDA approval in August 2023. Georgiamune also completed a $75 million Series A financing round during the same period. Notably,Samir Khleif, M.D., Founder and Chief Executive Officer of the company, will attend 2024.WuXi AppTec Global Forum, sharing insights into the development of new anti-cancer drugs.

19. Nested Therapeutics

One of the co-founders of Nested Therapeutics is Dr. Kevan Shokat, who has made significant contributions to overcoming the undruggability of KRAS., whose team's groundbreaking paper published in the top scientific journal *Nature* in 2013 ultimately paved the way for the development of Amgen's Lumakras (sotorasib), which targets the KRAS G12C mutation. Nested Therapeutics announced in October 2022 the completion of a $90 millionSeries A Financing。

Nested Therapeutics' platform comprises three major projects: mapping mutation clusters to their corresponding structural proteomes, identifying druggable "pockets" and their cancer-driving mechanisms, and designing optimized novel drugs for these druggable "pockets."NEST-1, the main project of Nested Therapeutics, is a molecular glue that does not degrade its target. It can target multiple components within the mitogen-activated protein kinase (MAPK) pathway and has demonstrated potential efficacy, tolerability, and central nervous system (CNS) activity as a single agent or in combination therapy across various RAS/MAPK-driven models. Although NEST-1 does not lead to the degradation of the target protein, it inhibits pathway activation by linking molecules within the pathway to other molecules, thereby suppressing disease progression.

20. Crossbow Therapeutics

Crossbow is a company dedicated to improving the lives of cancer patients by developing antibodies that mimic T-cell receptors (TCRs). The company's proprietaryT-Bolt Therapy is a new generation of immunotherapy with the potential to precisely target all cancer antigens, including previously untouchable cancer cell targets.。The company completed an $80 million financing in July 2023.Series A FinancingThe proceeds will enable Crossbow to advance the development of novel therapies.

Crossbow Therapeutics first identifies and validates cancer-specific antigens with potential. These antigen-specific peptides are presented on the surface of cancer cells through the major histocompatibility complex (MHC). The company then develops TCR-mimicking antibodies, which carry a TCR-mimicking protein domain on one part and a T-cell receptor-activating protein domain on another part. Such antibodies are expected to become off-the-shelf T-cell engagers and other immunotherapies.Briggs Morrison, Ph.D., CEO of Crossbow Therapeutics, Inc., will also participate in 2024.WuXi AppTec Global Forum, sharing the company's insights into the development of new cancer treatment models.

21. Ascidian Therapeutics

Ascidian Therapeutics is a company focused on replacing mutated exons at the RNA level to treat various human diseases. This technology has the potential to overcome the limitations of existing gene therapies, making it possible to target large genes or genes with multiple mutations. The company’s RNA exon editing platform combines advanced computational biology and high-throughput molecular biology screening to design an innovative RNA exon molecule.The designed RNA exon contains the correct RNA sequence and can interact with the target precursor messenger RNA (pre-mRNA), replacing the cell's own mutated exon through trans-splicing to achieve a therapeutic effect.

▲CEO of Ascidian TherapeuticsRomesh SubramanianDr.Insights Shared at the 2023 WuXi AppTec Global Forum

The company completed a $50 million financing in October 2023.Series A FinancingAt the 2023 WuXi AppTec Global Forum, Dr. Romesh Subramanian, the company's CEO, pointed out that technological development in the RNA field takes time. Taking the company’s core platform as an example, "20 years ago, the description of this technology was only a few lines long." The development in this field is based on the advancements of various technologies over the past 20 years, which have ultimately enabled RNA therapies to possess drug-like characteristics.Talking about the future of the RNA field, he said that this area is still very hot because "people want to make an impact on patients."

22. Delve Bio

Delve Bio Hopes to Bring a Genomics Revolution to Infectious Diseases.Delve Bio's Delve Neuro test samples all metagenomic components (RNA and DNA) in patient samples, enabling the simultaneous detection of bacteria, viruses, fungi, and parasites with a single test.The company completed a $35 million Series A financing round in June 2023.

23. Alterome Therapeutics

Alterome Therapeutics focuses on developing alteration-specific treatments to address the harm caused by cancer drivers.

The company completed over 35 million US dollars in funding in November 2022.Series A2 FinancingThe proceeds will be used to advance the development of three next-generation precision oncology programs and their associated product pipelines. These programs are designed based on Alterome's internal computational chemistry platform, "The Kraken."The Kraken platform provides precise insights into intermolecular interactions at the atomic level and enables predictions of ligand activity and binding modes, offering the company's team of medicinal chemistry experts the technical means to design novel therapies with high selectivity and specific targeting of cancer mutations.

24. LinusBio

LinusBio's platform is designed to identify which environmental factors individuals have been exposed to at different time points through precisely detected exposome biomarkers, and may potentially aid in disease diagnosis and progression monitoring.Using a strand of hair, time-exposure omics detection links genomics and the environment by mapping the dynamic changes in an individual's unique biological response to environmental exposures over time. Rather than simply assessing how many chemicals are present in the hair, it reveals how the measurements change over time and their interrelationship with environmental factors.

The company completed a USD 16 million Series A financing round in early 2023. The press release noted,Its early candidate diagnostic test, StrandDx-ASD, can assess the likelihood of children developing autism at birth with an accuracy rate of 80% to 90%, and help guide personalized treatment decisions.

25. Actio Biosciences

Actio Biosciences Develops New Therapies for Rare and Common Diseases Based on Underlying Biology Using Novel Genetic and Precision Medicine Platform Approaches.Actio Biosciences leverages its expertise in genetics, drug discovery, and data science to identify programs that minimize biological and technical risks, streamline the drug development process, and create highly effective, precisely targeted therapeutic drugs.

The company completed a $55 million financing in September 2023.Series A FinancingIts pipeline mainly includes a project targeting TRPV4, a high-value ion channel target identified by the company’s platform. Actio Biosciences plans to first evaluate this project for treating rare diseases caused by TRPV4 mutations, including Charcot-Marie-Tooth disease type 2C, characterized by myasthenia gravis, vocal cord paralysis, and respiratory complications, as well as severe bone disorders such as metatropic dysplasia.

26. i2o Therapeutics

i2o Therapeutics' technology platform is dedicated to converting injectable biologics and peptide drugs into oral therapies. It can protect biologics from damage caused by the gastrointestinal environment and assist them in crossing epithelial tissues.The company's R&D pipeline includes six therapies under development, including multiple long-acting GLP-1 receptor agonists and long-acting treatments for obesity and type 2 diabetes.

27. Initial Therapeutics

Initial Therapeutics, Inc. leverages its proprietary Selective Termination of Protein Synthesis (STOPS) platform to target protein targets that are difficult for traditional small-molecule drugs to address.The STOPS platform is based on the research of the company’s scientific founders, who discovered that small-molecule drugs can selectively interrupt protein translation at the ribosomal exit tunnel when specific proteins are first generated in their linear sequence.

The company completed a $75 million financing in May 2023.Series A FinancingUnlike intervention methods targeting mature proteins (such as targeted protein stabilization and protein degradation), the company's strategy bypasses the need to consider the activity of mature proteins and the issue of resolving their structure. Moreover, preventing protein synthesis may inhibit the formation of irreversible aggregates and other disease-related molecular pathological processes. From this perspective, the company’s approach may offer better therapeutic outcomes.

28. Entact Bio

Entact Bio's technology platform aims to develop drugs called ENTAC (enhancement-targeting chimeric). It canLinking beneficial target proteins with deubiquitinating enzymes (DUBs)DUBs can selectively shorten or remove ubiquitin chains linked to target proteins.Ubiquitin plays a broad role in protein function, thus enhancing the function of target proteins by shortening or removing ubiquitin.Whether the target protein is in short supply, located in the wrong position within the cell, or exhibits suboptimal activity, its function can be enhanced by designing an appropriate ENTAC to recruit DUB, ultimately restoring cellular health.

The design concept of traditional small-molecule drugs is to inhibit the function of proteins that cause diseases, rather than enhance the function of beneficial proteins. ENTAC has tremendous potential to expand the druggable space and address unmet medical needs. Entact Bio completed an $81 million financing in December 2022.Series A Financing。

29. Rezo Therapeutics

Rezo Therapeutics' technology platform integrates multi-layered data from genomics, proteomics, structural biology, chemical biology, and bioinformatics to achieve a more comprehensive and accurate understanding of disease mechanisms. These strategies help rapidly identify protein and gene interactions that cause diseases. The company completed a $78 million Series A financing round in November 2022.

Nevan Krogan, Ph.D., Co-founder and President of Rezo Therapeutics, Inc., will2024 WuXi AppTec Global ForumParticipated in the panel discussion "Delivering on Future Platform Solutions" to explore the impact of innovative technology platforms on new drug development.

30. Aether Bio

Aether Bio Focuses on Discovering Innovative Biocatalysts Using Generative Artificial Intelligence and Automation TechnologiesThe company's technology platform integrates high-throughput robotics, machine learning, and synthetic biology to rapidly match enzymes and chemical reactions, developing products with enhanced functions for use in the biopharmaceutical, home appliance, and automotive industries.

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