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, partnerships, R&D pipeline, growth potential, and innovation,BioSpaceSelect the Rising Stars in Life Sciences. This year's list includes30Jiaxin fully demonstrates the impact of new treatment models and innovative drug development strategies on new drug development., whose areas of focus are extensive, includingGene Therapy, Cell Therapy,RNATherapy、RadiopharmaceuticalsAnd innovative small moleculesetc.DifferentMode.

1. Rapport Therapeutics

Rapport Therapeutics' precision medicine development strategy leverages 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 financing 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，Its co-founder, Crystal Mackall, MD, the founding director of the Stanford Cancer Cell Therapy Center and a pioneer in T-cell research, will attend in 2024.WuXi AppTec Global Forum, Participate in panel discussions.

4. Aera Therapeutics

Aera Therapeutics, founded by gene-editing pioneer Professor Feng Zhang, aims to utilize a human protein named PEG10, which can self-assemble into structures resembling viral capsids and can be used to deliver RNA, DNA, and gene-editing systems. Last year, the company completed its Series A and B financing rounds, raising $193 million.This vector has lower immunogenicity compared to viral vectors, which 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 did so in May of last year.AnnounceCompleted seed round, Series A, and Series B financing, with a total amount of $109 million.

6. ReNAgade Therapeutics

ReNAgade Therapeutics is an RNA therapy development company distinguished by its delivery platform and proprietary tools. This platform combines 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 various 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。Dr. Giuseppe "Pino" Ciaramella, the company's co-founder, CEO and President, will also attend in 2024.WuXi AppTec Global Forum, jointly discuss how emerging treatment models are leading the industry into a new round of transformation and breakthroughs.

8. Abdera Therapeutics

Abdera Therapeutics focuses on developing targeted antibody-based delivery of 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 molecular proteins, thereby enhancing therapeutic efficacy.Its DLL3-targeted small cell lung cancer therapy is planned to submit an IND in 2024. Ms. Lori Lyons-Williams, the company's president and CEO, mentioned during the 3rd edition of WuXi AppTec’s “BOLD: Innovation Leads, Brave the Future” series: "The power of radiopharmaceuticals lies in their highly potent and targeted payloads."We believe that in the next decade 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. The principle is that when microbubbles are ruptured by ultrasound, the released energy creates small pores in the cell membrane, facilitating 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 has 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 program, 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 seen with other CD47-targeting drugs. Second, it has a higher level of purity, enhancing its affinity for the CD47 target and thus requiring a lower dosage. Furthermore, it only needs to penetrate the blood vessel walls without entering the tumor, so a small amount of the drug can show effects in cases of atherosclerosis. The company completed$145 millionSeries A Financing。Dr. Pavan Cheruvu, CEO and President of Bitterroot Bio, will attend 2024.WuXi AppTec Global Forum, Discuss the latest industry R&D and breakthrough trends.

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. Belharra’s approach is to directly analyze proteins within cells rather than studying only purified proteins or static snapshots from structural research. This method allows Belharra to more accurately understand 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 currently discovered over 4,000 new small molecule-protein pairs, including those with 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 rigorous 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 biomedical 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 tissues.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, M.D., co-founder and CEO of the company, said at the recent conclusion of the "BOLD: Innovation Leads, Brave the Future" Series 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 dose of targeted radiation.This advancement has greatly promoted the development of the 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 complexities 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 Therapeutics Aims to Develop Innovative Covalent Small Molecule Drugs, with Initial R&D Pipeline Focused on Immunology. The Company’s Goal is to Develop Therapeutic Agents Using Covalent Chemistry for Targets Already Validated by Human Genetics or Clinically.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 against targets with very low abundance within their natural 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 raised $100 millionFinancing。

16. Bonum Therapeutics

Bonum focuses on the development of 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, which activate the activity of the therapeutic molecule upon binding to the target. Its sensor protein domain can bind to peptides, proteins, or metabolites. Besides cancer treatment, this technology has exciting application potential in treating autoimmune diseases, metabolic diseases, and pain relief.The company completed a $93 million financing in November 2022.Series A Financing。

17. Juvena Therapeutics

Juvena Therapeutics' platform combines databases that reveal the connection between secreted proteins and specific diseases, computational screening technologies, experimental screening in 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 its biologics pipeline targeting chronic and age-related diseases.

18. Georgiamune

Georgiamune focuses on reprogramming immune signaling pathways to redirect the immune system to combat diseases.The 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 lead 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 simultaneously completed a $75 million Series A financing round. Notably,Samir Khleif, M.D., Founder and Chief Executive Officer of the company, will attend 2024.WuXi AppTec Global Forum, sharing insights in 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 the target and can target many components within the Mitogen-Activated Protein Kinase (MAPK) pathway. In numerous RAS/MAPK-driven models, it demonstrates potential excellent efficacy, tolerability, and Central Nervous System (CNS) activity as either a single agent or in combination therapies. Although NEST-1 does not lead to the degradation of the target protein, it inhibits the activation of signaling pathways by linking molecules within the pathway to other molecules, thereby achieving the goal of 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, 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 with 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. inInsights 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. Using the company’s core platform as an example, “20 years ago, there were only a few lines describing this technology.” The advancement in this field is based on the development of various technologies over nearly 20 years, which has 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 Aims to Bring 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 USD 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 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 precise detection of exposome biomarkers, potentially aiding in disease diagnosis and monitoring progression.Using a strand of hair, time-exposure omics detection links genomics and the environment by mapping the dynamic changes of an individual's unique biological responses to environmental exposures over time. Rather than simply assessing how many chemicals are present in the hair, it reveals how the measured values change over time and their interrelationship with environmental factors.

The company completed a $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 deliver 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 the treatment of 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 diseases such as metatropic dysplasia.

26. i2o Therapeutics

i2o Therapeutics' technology platform is dedicated to transforming 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 be used to selectively interrupt the translation of proteins when their linear sequence is first generated in the ribosomal exit tunnel.

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 difficult-to-reverse aggregates and other disease-related molecular pathological processes. From this perspective, the company’s approach may offer better therapeutic effects.

28. Entact Bio

Entact Bio's technology platform is designed 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 disease, rather than enhance the function of beneficial proteins. ENTAC has enormous 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 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.