Recently, well-known industry media Endpoint News released the "Biopharma's Most Exciting Startups in 2024" list, which includes 11 biotechnology companies. These emerging research fields include gene editing, immunotherapy, antibody conjugated drugs (ADCs), weight loss therapy, and multiple other directions. This article will introduce these 11 rising stars (listed in no particular order) to readers, based on this ranking and official information from various companies.

Lifordi Immunotherapies is a biotechnology company dedicated to developing ADC therapies for the treatment of autoimmune and inflammatory diseases. Lifordi was founded in 2023 and has secured $70 million in Series A funding from ARCH Venture Partners, 5AM Ventures, and Atlas Venture, with the goal of advancing the main candidate drug LFD-200 into clinical trials and obtaining preliminary data by the end of 2025. Lifordi's main ADC therapy LFD-200 targets myeloid cells and lymphocytes by targeting highly internalized cell surface facial mask proteins. This therapy has proved its efficacy in a variety of preclinical disease models. The experimental results showed that the drug has a short serum half-life, a long residence time in immune cells, and can persistently exert immunosuppressive function in these cells without producing systemic delivery related toxicity. Lifordi's ADC delivery platform can be used to target innate and adaptive immune cells, carrying diverse payloads including small molecules, antisense oligonucleotides (ASO), and siRNA. This platform can be applied to develop innovative therapies for treating autoimmune and inflammatory diseases in various fields such as rheumatology, gastroenterology, pulmonology, and dermatology.

Mirador Therapeutics announced in March this year that it had moved out of stealth mode and completed over $400 million in funding. The company was founded by Mr. Mark C. McKenna and led by several former executives of Prometheus Biosciences (Prometheus was acquired by Merck for $10.8 billion last year). Mirador aims to revolutionize precision medicine for immune-mediated inflammatory and fibrotic diseases by rapidly advancing multiple projects through the use of its proprietary Mirador360 development engine. Mirador focuses on developing potential "first in class" or "best in class" precision medicine drugs. Mirador's proprietary Mirador360 platform combines the latest breakthroughs in human genetics and cutting-edge data science, capable of analyzing the molecular profiles of millions of patients to discover and validate genetic information related to immune fibrosis diseases, identify new therapeutic targets, elucidate interactions between targets, and discover optimal target pairings for potential combination therapies. In addition, Mirador360 can assist Mirador in developing diagnostic tests and stratifying patient populations for precise clinical development.

Formation Bio announced in June this year that it had completed a $372 million Series D funding round. The company aims to build a technology platform empowered by artificial intelligence (AI) to accelerate various aspects of drug development and clinical trials. The company plans to allocate the new funds towards two main goals: introducing candidate drugs and expanding its AI capabilities. In terms of introducing candidate drugs, Formation Bio plans to introduce projects in the clinical development stage from biotechnology and pharmaceutical partners. In terms of AI enabled drug development, through its technology driven operational model, Formation Bio is able to conduct clinical trials faster and more efficiently, simplifying activities such as study initiation, participant recruitment, data management, database locking, and clinical trial closure. Based on its technology, data, and operational foundation, the company is able to quickly integrate large language models (LLMs), AI models, and applications into the platform. Formation Bio partnered with Sanofi in May this year to jointly develop AI driven software aimed at accelerating drug development and delivering innovative drugs to patients more efficiently.

Xaira Therapeutics announced its official launch in April of this year. The company was co incubated by ARCH Venture Partners and Foresite Labs, and received over $1 billion in startup funding. According to a report by Endpoint News at the time, this was one of the startups that received the highest funding this year. Xaira is a comprehensive biotechnology company dedicated to using artificial intelligence to drive advances in life sciences and change the way diseases are treated. The company aims to rethink the drug discovery and development process end-to-end by gathering top talent. The company mainly covers three core areas: advanced machine learning research, large-scale data generation, and powerful therapy development capabilities. The company is star studded, with Professor David Baker, a pioneer in artificial intelligence driven protein folding and design, as one of its co founders, and Dr. Marc Tessier Lavigne, former Chief Scientific Officer of Genentech, joining the team as CEO.

Yilian Biotechnology announced a partnership with Roche earlier this year. Roche has obtained exclusive rights to the development, manufacturing, and commercialization of YL211, a new generation ADC product targeted by C-MET from Yilian Biotech, on a global scale. According to the agreement, Yilian Biotechnology will collaborate with Roche China Innovation Center (CICoR) to promote the Phase 1 clinical trial of YL211, and assign Roche to be responsible for further development and commercialization worldwide. Yilian Biotechnology has received a down payment and recent milestone payments of 50 million US dollars, with an additional potential milestone payment of nearly 1 billion US dollars. In May of this year, Yilian Biotechnology announced that the first phase 1 clinical trial of YL211 in the United States had completed administration to a subject. In October 2023, Yilian Biotechnology and BioNTech reached a strategic cooperation and global licensing agreement on YL202/BNT326, an ADC project targeting HER3. In May of this year, Yilian Biotechnology and BioNTech reached a new strategic partnership. BioNTech will have the exclusive choice and corresponding global exclusive license to develop ADC products targeting specified cutting-edge innovative targets using the Yilian TMALIN antibody conjugated drug technology platform. According to the terms of the new agreement, Yilian Biotechnology will receive a prepayment of $25 million and is eligible for up to $1.8 billion in development, regulatory, and commercialization milestone payments.

Iambic Therapeutics announced in June this year the completion of a $50 million Series B expansion funding. The funding will be used to advance Iambic's clinical and preclinical projects, including IAM1363, a highly selective HER2 inhibitor that can cross the blood-brain barrier. It can inhibit wild-type and HER2 receptors carrying carcinogenic mutations, and is currently in phase 1/1b research. The funds obtained will also be used to develop a potential "first in class" CDK2/4 inhibitor, aiming to expand the treatment window and solve the drug resistance problem in solid tumors such as breast cancer. Iambic's candidate drugs were discovered using its AI protein structure prediction and overall drug design model. These technologies are integrated into a closed-loop, automated, high-throughput platform for biological and chemical experiments, providing new biological insights from thousands of molecular designs every week, directly informing their AI models.

Ascidian focuses on treating various human diseases at the RNA level by replacing exons carrying mutations. This technology has the potential to break through the limitations of existing gene therapies, making it possible to target large genes or multiple mutated genes. The company's RNA exon editing platform combines advanced computational biology and high-throughput molecular biology screening to design an innovative class of RNA molecules. The designed RNA contains the correct RNA sequence and can interact with the target messenger RNA precursor (pre mRNA) by trans splicing to replace the mutated exons carried by the RNA itself, achieving therapeutic effects. The company's fastest progressing research and development project is the investigational therapy ACDN-01 for ABCA4 retinal disease. Its IND application has been approved by the US FDA, and the related Phase 1/2 clinical trial was launched in June this year. The company stated that this is the first RNA exon editing therapy to enter the clinical development stage. Recently, the company reached a research collaboration and licensing agreement with Roche worth approximately $1.8 billion, in which the two companies will jointly discover and develop RNA exon editing therapies for neurological diseases. The company completed a $50 million Series A financing in October 2023. At the 2023 WuXi AppTec Global Forum, Dr. Romesh Subramanian, the then CEO of the company, pointed out that technology development in the RNA field requires time. Taking its core platform as an example, "20 years ago, the description of this technology was only a few short lines." However, the development of this field is based on the development of multiple technologies over the past 20 years, ultimately giving RNA therapy the characteristics of a drug. When it comes to the future of RNA, he said that this field is still very hot because "people want to have an impact on patients".

Capstan is committed to utilizing its targeted lipid nanoparticle (tLNP) technology platform to deliver RNA to specific cells in patients' bodies and reprogram the cells. In March of this year, the company completed a Series B financing of $175 million, which will be used to advance the early clinical concept validation of its flagship chimeric antigen receptor T cell (CAR-T) candidate therapy CPTX2309 in autoimmune diseases, and further develop Capstan's targeted lipid nanoparticle pipeline. CPTX2309 is a product of Capstan's tLNP platform, which delivers mRNA payloads encoding chimeric antigen receptors targeting CD19 to CD8 expressing T cells, effectively generating CAR-T cell therapy directly in vivo. The goal of this method is to achieve immune system reset and avoid the challenges of traditional in vitro CAR-T cell therapy by rapidly and deeply clearing B cells from blood and lymphoid tissues. At the 2023 WuXi AppTec Global Forum, Dr. Shawver, CEO of the company, stated that in the field of gene therapy, the industry has recognized the limitations of viral vectors, and therefore non viral vectors, including LNP, will have a place in future gene therapy development. For LNP carrier technology, how to specifically deliver LNP to different types of cells is a problem that needs to be solved. Capstan's tLNP technology platform is expected to solve the problem of specific delivery by decorating LNP with antibodies or antibody fragments that bind to specific receptors.

Seaport Therapeutics focuses on advancing the development of innovative neuropsychiatric drugs driven by its proprietary Glyph technology platform. The platform aims to improve the oral bioavailability of drugs, avoid first pass metabolism, reduce liver toxicity and other side effects, in order to advance active drugs that were previously put on hold due to these limitations. The fastest progressing candidate drug for Seaport is SPT-300, an oral prodrug of allopregnanolone that is being developed for the treatment of anxiety and depression. Belgnanol ketone is an endogenous pregnane class neurosteroid that has demonstrated therapeutic benefits in a range of neurological and psychiatric disorders, but it is only approved for intravenous injection, which limits its clinical use. Using the Glyph platform, SPT-300 retains the activity and efficacy of endogenous sex pregnenolone and is used orally. In the Phase 2a clinical trial, SPT-300 has obtained conceptual validation in a clinical anxiety model conducted in healthy volunteers. In April of this year, the company announced the completion of a $100 million Series A financing. The company was co founded by Daphne Zohar, founder of PureTech Health, and Dr. Steven Paul, former CEO of Karuna Therapeutics. The leadership team has extensive experience in developing drugs for neurological and psychiatric disorders. Karuna Therapeutics has been acquired by Bristol Myers Squibb for $14 billion, and its developed schizophrenia therapy Cobenfy has just received FDA approval for marketing.

Metsera is committed to developing a product portfolio for the treatment of obesity, as well as obesity and metabolic related diseases. In April of this year, the company announced the completion of a $290 million financing. Metsera's new generation of injectable and oral drug development project portfolio is derived from the company's proprietary library of over 20000 intestinal hormone peptides and peptide/antibody conjugates. The company focuses on multiple new generation targets and combination therapies. Recently, the company announced that its ultra long acting weight loss therapy MET-097 has achieved significant and long-lasting weight loss effects in phase 1 clinical trials. After 36 days of treatment, the subjects experienced a weight loss of 7.5%, and their pharmacokinetic characteristics supported a potential monthly dosing regimen. Metsera plans to launch the Phase 2b clinical trial of MET-097 in the fourth quarter of 2024. MET-097 is an under development ultra long acting subcutaneous GLP-1 receptor agonist. In addition, the company's R&D pipeline also includes research and development projects such as dual receptor agonists of pancreatic amyloid peptide/calcitonin (DACRA), single-molecule GGG (targeting GLP-1, glucose dependent insulinotropic polypeptide [GIP], glucagon] therapy, etc. These therapies have been carefully designed to have a potential "best in class" duration of efficacy and can form multiple combination therapies.

Cardurin is committed to developing a new generation of heart disease therapies. The company announced in July this year that it had completed a $260 million Series B financing, primarily to support clinical trials of its two flagship drug candidates. They are potential "first in class" phosphodiesterase-9 (PDE9) inhibitors for treating heart failure and calcium/calmodulin dependent protein kinase II (CaMKII) inhibitors with broad therapeutic potential. Currently, its flagship PDE9 inhibitor CRD-750 is being evaluated in two phase 2 clinical trials involving a total of 640 heart failure patients. The CaMKII inhibitor CRD-4730 is currently undergoing phase 2 clinical trials for the treatment of catecholamine polymorphic ventricular tachycardia (CPVT) patients. Dr. Michael Mendelsohn, founder of Carduria Pharmaceuticals and internationally renowned cardiovascular scientist, stated at the 2022 WuXi AppTec Health Industry Forum that a key obstacle in cardiovascular drug development is attempting to design well thought out clinical trials. Focusing on patient phenotype refinement and novel biomarkers will be one of the core aspects of clinical trial design.

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