NIDO-361

Pictured: Map of NextGen Class of 2024/BioSpace BioSpace is proud to present its NextGen Bio Class of 2024, a list of the hottest new life sciences companies in the United States. To come up with this list, BioSpace identified companies that launched between September 2022 and September 2023 with Series A funding. They were then assessed using several different criteria including finance, partnerships, pipeline, growth potential and innovation. After awarding points for each category, the BioSpace editorial team ranked its top 30. The impressive group of startups that make up BioSpace’s NextGen Class of 2024 is already making an impact on the industry. We will continue to keep a close eye on these companies as we move into the new year. 1. Rapport Therapeutics Launched: March 2023 Location: Boston, MA, and San Diego, CA Series A: $100M Series B: $150M Notable: Good rapport won this startup top billing on our list. The product of a partnership between Third Rock Ventures and Janssen Neuroscience, Rapport’s first program focuses on targeting drug-resistant seizure disorders and is in Phase I development. Differentiator: Specificity is what sets Rapport apart, according to CEO Abe Ceesay, who spoke with BioSpace in March 2023. Current therapies for neurological disorders are nonspecific in nature, Ceesay said, while Rapport’s technology, which uses receptor-associated proteins (RAPs), has the potential to deliver highly specific therapies that are both more effective and better tolerated. The approach allows “unprecedented precision” in targeting receptors in the specific neuroanatomical regions underlying neurological disorders, according to the Series A announcement. One More Thing: Not six months after announcing a $100 million Series A, Rapport came back with $150 million in Series B financing. 2. Apogee Therapeutics Launched: December 2022 Location: Waltham, MA Series A: $20M Series B: $149M Notable: In August 2023, Apogee dosed the first participants in a Phase I trial of lead candidate APG777, which is being developed to treat atopic dermatitis and other inflammatory diseases. APG777 is also in preclinical development for asthma. Differentiator: Apogee is building antibodies from scratch, CEO Michael Henderson told BioSpace at the time of the company's Series B announcement in December 2022. “We are going to build brand new, novel antibodies that will provide a meaningful step change to what these patients currently have,” he said. One More Thing: Apogee, the first spinout from Paragon Therapeutics, was one of just a few biotechs to an initial public offering (IPO) in 2023. 3. CARGO Therapeutics Launched: March 2023 Location: San Mateo, CA Series A: $200M Notable: CARGO is another member of the select 2023 IPO club. The quickly moving startup announced its own filing in November 2023, seeking more than $315 million. The company plans to use around $220 million of the proceeds from the IPO to fund Phase II trials of its lead candidate CRG-022, an autologous CD22 CAR T cell therapy designed for patients whose large B-cell lymphoma (LBCL) is relapsed or refractory to CD19 CAR T cell therapies. Differentiator: CARGO already has data in hand from a Phase I trial showing that treatment with CRG-022 led to durable complete responses in greater than 50% of LBCL patients in this population. One More Thing: CRG-022 has been granted Breakthrough Therapy Designation by the FDA. 4. Aera Therapeutics Launched: February 2023 Location: Cambridge, MA Series A & B: $193M Notable: Aera launched into the sizzling hot field of gene therapy delivery with foundational technology from the lab of world-renowned scientist Feng Zhang of the Broad Institute of MIT and Harvard and the University of Utah’s Jason Shepherd. Differentiator: Pedigree. Aera was founded by Zhang and ARCH Venture Partners. Zhang is joined by former Alnylam Oncology Head Akin Akinc, who serves as CEO, and John Maraganore, the founding CEO of Alnylam Pharmaceuticals, who serves as Aera’s board chair. One More Thing: Aera will “explore a variety of genetic medicine modalities” and “prioritize applications based on patient impact,” Akinc told BioSpace in an email at the time of the Series A and B announcement. 5. Nido Biosciences Launched: May 2023 Location: Watertown, MA Series A, B & Seed: $109M Notable: Nido was founded through the 4:59 Initiative at 5AM Ventures. The company’s first clinical-stage candidate, NIDO-361, is in Phase I trials for Spinal and Bulbar Muscular Atrophy (SBMA)—also known as Kennedy’s disease—a rare, inherited neuromuscular disorder affecting men that results in a loss of skeletal muscle and motor neuron function. The disorder is caused by a genetic mutation of the androgen receptor. Differentiator: In addition to NIDO-361, Nido is leveraging a functional genomics discovery platform based on human cell lines that uses tailored screens to identify novel therapeutic targets for myriad neuromuscular and neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS) and frontotemporal disorders (FTD). One More Thing: Eli Lilly, which has a deep emphasis on neuroscience, participated in Nido’s Series A funding round. 6. ReNAgade Therapeutics Launched: May 2023 Location: Cambridge, MA Series A: $300M Notable: The past three years have made clear the potential of RNA, but the aptly named ReNAgade Therapeutics wants to push the envelope and deliver RNA medicines to previously inaccessible tissues and cells. Differentiator: Experience. ReNAgade CEO Amit Munshi was most recently the head of Arena Pharmaceuticals, which under his stewardship grew from a $300 million company into a late clinical-stage firm with nearly 40 programs before being acquired by Pfizer for $6.7 billion. Munshi is joined by CSO Peter Smith, whose resume includes leadership roles at Alnylam and Moderna. One More Thing: Not only did ReNAgade launch with a cool $300 million, but it had also already established a joint venture with circular RNA startup Orna Therapeutics—BioSpace Class of ’22—to combine their respective platforms. A subsequent Orna-Merck collaboration is leveraging tech developed under this joint venture. 7. Orbital Therapeutics Launched: April 2023 Location: Cambridge, MA Series A: $270M Notable: Orbiting in the red-hot RNA space, this startup is a spinout of Beam Therapeutics and counts John Maraganore, the founding CEO of Alnylam Pharmaceuticals, as a co-founder. Maraganore is chairman of Orbital’s board of directors. Differentiator: Orbital is building what it calls a first-in-kind platform that combines existing and novel RNA technologies and delivery mechanisms. And it would seem the company has the cash and experience to do it. One More Thing: Co-founder and CEO Giuseppe Ciaramella is a busy man, also serving as president of Beam. Ciaramella was previously CSO of the infectious diseases division at Moderna. 8. Abdera Therapeutics Launched: April 2023 Location: Menlo Park, CA, and Vancouver, BC Series A & B: $142M Notable: The radiopharmaceuticals space quietly picked up speed in 2023, with Eli Lilly’s $1.4 billion acquisition of Point Biopharma, RayzeBio’s $311 million IPO and Abdera Therapeutics’ $142 million in combined Series A and B funds. Differentiator: Balance. Abdera’s website notes that radiopharmaceuticals using small ligands often fail to reach therapeutic levels inside tumors and can cause toxicities, while antibodies can limit tumor penetration and lead to systemic exposure. Abdera’s proprietary ROVEr platform allows it to strike an optimal balance between the two approaches, the company claims, with antibody-based medicines that bear a high-affinity antigen-binding domain to seek out and specifically target cancer cells. One More Thing: Abdera’s lead asset, ABD-14, is a delta-like ligand 3-targeting molecule going after small cell lung cancer. 9. SonoThera Launched: December 2022 Location: South San Francisco, CA Series A: $60.75M Notable: Eli Lilly and J&J are among the investors that believe in the potential of SonoThera’s ultrasound-guided nonviral gene delivery platform, as the two companies kicked in funds for the startup’s Series A. Differentiator: Microbubbles. Whereas traditional approaches use viral vectors to deliver a therapeutic gene, SonoThera houses its payload in a microbubble, which is then injected into the bloodstream. The microbubble is guided to the target site using an FDA-cleared ultrasound device and a proprietary acoustic profile. This approach could allow the company to avoid the immunogenicity and high costs associated with viral vectors. One More Thing: As of February, SonoThera had two new microbubble products to use in the development of its platform after inking an exclusive license for GE HealthCare’s Optison and Sonazoid. 10. Bitterroot Bio Launched: June 2023 Location: Palo Alto, CA Series A: $145M Notable: The cardiovascular space experienced a resurgence in 2023, including the $145 million in Series A funds dropped into the coffers of Bitterroot Bio, a startup focused on the subspecialty of cardio-immunology. Differentiator: Bitterroot’s first program targets the CD47/SIRPα pathway. While the anti-CD47 approach to cardiovascular pathology has yet to be tested in humans, blocking CD47 restored the function of macrophages in mice and facilitated the clearance of plaques and reversal of atherosclerosis, according to a 2016 study published in Nature. One More Thing: Co-founder Irving Weissman is credited with the discovery of how tumors use the CD47 protein to evade the immune system. 11. Belharra Therapeutics Launched: January 2023 Location: San Mateo and San Diego, CA Series A: $130M Notable: Backed by Versant Ventures and Roche’s Genentech, Belharra Therapeutics launched with a nine-figure funding round and a drive to advance its chemoproteomics platform. Differentiator: Belharra’s chemoproteomics platform does not require specific amino acid residues on a protein to find and label a proteome. When the company announced its launch, it said its platform could counter the challenges in chemoprotemoics. One More Thing: So far, the company plans to set its discovery sights in the oncology and immunology spaces, but it has also netted partnerships with Genentech and Scripps Research. 12. Kate Therapeutics Launched: June 2023 Location: San Diego, CA Series A: $51M Notable: Kate Therapeutics is a biotech looking to develop AAV-based treatments for complex heart and muscle conditions. Differentiator: Kate Therapeutics separates itself from the pack with its DELIVER platform, which uses directed evolution, RNA-based selection of capsid variants and machine learning to learn and analyze in vivo models. Kate has produced a class of capsids called MyoAAV, developed by Sharif Tabebrodbar, Kate’s CSO and co-founder. One More Thing: Kate hit the ground running in partnerships with bigger pharma companies. Astellas inked an exclusive license to develop, produce and commercialize one of Kate’s candidates, KT430, designed to treat X-linked myotubular myopathy. 13. Convergent Therapeutics Launched: May 2023 Location: Cambridge, MA Series A: $90M Notable: The startup is seeking to develop radiopharmaceutical-based therapies to treat cancers, including prostate cancer. Differentiator: Convergent claims that its lead candidate in targeting prostate cancer, CONVA-α, is different from other radioactive medicines. Using the radionuclide 225Ac, the alpha particles can deliver radiation over short distances and reduce exposure to healthier tissues and cells nearby, according to the company. In a Phase I trial of the candidate, 44% of patients with metastatic castration-resistant prostate cancer experienced a decline of prostate-specific antigen levels by more than 50% after a single dose. One More Thing: The company attracted big-name funders such as RA Capital Management and OrbiMed. 14. Cajal Neuroscience Launched: November 2022 Location: Seattle, WA Series A: $96M Notable: Backed by Bristol Myers Squibb, Lux Capital and The Column Group, Cajal Neurosciences plans to combine human genetics and multi-omics, whole brain imaging and functional genomics to find potential drug targets. Differentiator: A spokesperson at Cajal told BioSpace in late 2022 when the company launched that the discovery outfit uses computational genetic techniques to “identify the precise mechanism by which genetic variation mediates disease.” The company plans to build a discovery platform that allows researchers to see what is happening in a degenerating brain, leading to potential target points. One More Thing: Cajal is named after and inspired by Spanish neuroscientist Santiago Ramon y Cajal, who helped map cellular content and connectivity in the brain. 15. Matchpoint Therapeutics Launched: October 2022 Location: Cambridge, MA Series A: $100M Notable: Matchpoint Therapeutics came on the scene with backing from Sanofi and Vertex and is focused on developing precision small-molecule medicines. Differentiator: Its Advanced Covalent Exploration platform uses several tools such as chemoproteomics to identify new covalent binders to disease-causing proteins as well as machine learning to find targets and build out its library of covalent compounds. One More Thing: Matchpoint’s financing is enough to see the company through 2025. 16. Bonum Therapeutics Launched: November 2022 Location: Seattle, WA Series A: $93M Notable: After Roche acquired Good Therapeutics, the latter’s leadership team formed Bonum Therapeutics, which is developing a new class of “context-dependent therapeutics” that are only active when bound to a target molecule. Differentiator: Bonum focuses on the regulation of cytokines such as IL-12, IFN-alpha and TGF-beta, with an immune-oncology focus, but the company’s technology can also have applications in other sectors. The therapeutics themselves have antibody-binding domains that can act as sensors. When bound to a target, the molecules become active. One More Thing: Founder John Mulligan told BioSpace in 2022 that when the deal with Roche closed, the assets that were not transferred over were folded into the new company. The company is “the same as Good Therapeutics minus one program.” 17. Juvena Therapeutics Launched: November 2022 Location: Redwood City, CA Series A: $41M Notable: Launched with backing from Mubadala Capital and Horizons Ventures, Juvena Therapeutics aims to target chronic and age-related diseases. Differentiator: Juvena uses artificial intelligence to look for proteins secreted by regenerative stem cells. It plans to use the identified proteins as drug agents and develop biologics that can target their pathways as well. One More Thing: Juvena is still in the very early stages but already has several candidates in its pipeline for diseases such as myotonic dystrophy, sarcopenic obesity and obesity. 18. Georgiamune Launched: August 2023 Location: Gaithersburg, MD Series A: $75M Notable: Backed by several investment firms, including Mubadala Capital, Alexandra Venture Investments and Catalio Capital, among others, Georgiamune already has already picked up FDA clearance for an IND for one of its candidates, a dual-function monoclonal antibody called GIM-122 to fight cancer. Differentiator: Georgiamune’s approach centers on reprogramming immune signaling pathways to find ways to redirect the immune system to counter disease. Its strategy is to activate or down-modulate the immune system to fight cancer or autoimmune diseases. One More Thing: The company has four additional cancer candidates besides GIM-122 and two in unspecified autoimmune diseases. 19. Nested Therapeutics Launched: October 2022 Location: Cambridge, MA Series A: $90M Notable: Nested is focused on discovering and developing precision therapies to treat cancer and has received backing from prominent financial names such as Goldman Sachs Asset Management and Cowan Healthcare Investments. Differentiator: Nested’s platform can map mutational clusters onto the structural proteome, find “druggable pockets,” and design new drugs for those pockets, according to the company. One More Thing: Nested is already producing assets, with its lead compound known as NEST-1, a non-degrading dual molecular glue targeting the MAPK pathway. 20. Crossbow Therapeutics Launched: July 2023 Location: Cambridge, MA Series A: $80M Notable: Eli Lilly and Pfizer Ventures have served as some of the investors in Crossbow’s Series A round. The company claims that its cancer therapies have the power and precision of a crossbow. Differentiator: Crossbow says that its development process starts with finding a target and then uses TCR-mimetic antibodies to make an off-the-shelf T-cell engager and other immunotherapies. According to the company, its T-Bolt molecules can be adopted across a broad range of cancers. One More Thing: Apart from big names attached to the raise, the company was founded, seeded and incubated at MPM BioImpact. 21. Ascidian Therapeutics Launched: October 2022 Located: Boston, MA Series A: $50M Notable: When explaining the problem Ascidian aims to fix, CEO Romesh Subramanian described an editor’s worst nightmare. Imagine opening a book that has been published and finding spelling and grammatical errors throughout, Subramanian said in an interview with BioSpace at the time of the Series A financing. Instead of correcting each mistake manually, he said, it would be more efficient to precisely fix whole chapters of the book. “That’s what we can do with disease. What we essentially do is use RNA to splice in and replace mutated exons with wild-type exons. If there are multiple mutations, we can drop in a large set of exons and correct it.” Differentiator: Unlike gene therapies that target DNA, Ascidian’s approach makes these exon swaps in the RNA transcripts that encode proteins. Its lead program targets ABCA4 retinopathy, including Stargardt disease. One More Thing: Ascidian takes its name from the sea squirt. To grow from larvae to adults, ascidians re-engineer their transcriptome through RNA trans-splicing and alternative splicing. 22. Delve Bio Launched: June 2023 Located: San Francisco, CA Series A: $35M Notable: Delve was spun out of the University of California, San Francisco. Differentiator: Delve wants to bring the genomics revolution to infectious disease. The company’s test, Delve Neuro, samples all of the metagenomic content—RNA and DNA—within a patient’s sample to simultaneously detect bacteria, viruses, fungi and parasites. One More Thing: Delve’s Series A financing was led by Perceptive Xontogeny Venture Fund II, which was joined in the round by Section 32 and GV. 23. Alterome Therapeutics Launched: November 2022 Located: San Diego, CA Series A (extended): $99M+ Notable: This San Diego–based startup is going after particularly evasive cancer targets in the “alterome,” which is made up of all the disease-driving alterations in genes. Differentiator: Alterome’s computational chemistry platform, The Kraken, offers precise, atomic-level insights into molecular interactions, enabling ligand activity and binding mode predictions, according to the company’s website. One More Thing: Scientific Advisory Board member Paul Workman is a precision cancer treatment pioneer, having developed the concept of the Pharmacological Audit Trail for biomarker-led drug discovery and development. 24. LinusBio Launched: January 2023 Located: New York Series A & A2: $24M Notable: LinusBio is going after some pretty lofty targets with its exposome sequencing platform, including autism and ALS. Differentiator: LinusBio’s platform introduces precision exposome biomarkers to identify a person’s various environmental exposures and potentially help diagnose diseases and monitor progression and serve as objective endpoints in clinical trials. One More Thing: An early diagnostic candidate, StrandDx™-ASD, can assess the likelihood of autism at birth with 80% to 90% accuracy and help inform personalized treatment decisions, according to the Series A announcement. 25. Actio Biosciences Launched: September 2023 Location: San Diego, CA Series A: $55M Notable: Genetics and precision medicine are a common refrain among the companies on this year’s list, but Actio is launching its development efforts with rare diseases, including Charcot-Marie-Tooth disease type 2C. Differentiator: With its proprietary tech and deep genetics expertise, Actio is developing novel therapies that target shared underlying biology in both rare and common diseases. One More Thing: “Defining a high-impact drug target for a common disease with a heterogenous population has a very low probability of success and has long been a major challenge for drug discovery,” Actio co-founder and CEO David Goldstein said in a statement at the time of the Series A announcement. By starting with rare diseases, the company hopes to both help patients in these indications and de-risk and guide therapeutic expansion to more common conditions, Goldstein said. 26. i2o Therapeutics Launched: August 2023 Located: Cambridge, MA Series A: $46M Notable: i2o’s therapeutic focus couldn’t be hotter: The startup is researching a GLP-1 asset for type 2 diabetes in preclinical studies. This drug candidate, and several others, came with i2o’s acquisition of Intarcia Therapeutics. Differentiator: i2o’s proprietary ionic liquids platform seeks to overcome two barriers to oral administration of biologics and peptides by protecting them from the harsh conditions in the gastrointestinal tract and assisting large molecules in crossing the epithelial lining. One More Thing: i2o attracted the early attention of Sanofi Ventures, which took part in the company’s $4 million seed round. Both Sanofi and J&J are development partners. 27. Initial Therapeutics Launched: May 2023 Located: South San Francisco, CA Series A: $75M Notable: Initial Therapeutics is another startup that bills itself on attempting to drug undruggable targets, in this case using selective termination of protein synthesis, or STOPS, to discover therapeutics. Differentiator: Initial’s STOPS platform comprises a “bespoke suite” of technologies and capabilities, according to the company’s website. This allows the team to discover small molecules to intercept the translation of target proteins, Initial states in its Series A announcement, adding that its strategy “circumvents the need to accommodate the cellular activity of the fully formed protein or to structurally solve for docking.” One More Thing: Initial was launched by the life sciences venture capital firm Apple Tree Partners. 28. Entact Bio Launched: December 2022 Located: Watertown, MA Series A: $81M Notable: Entact Bio isn’t targeting disease-causing proteins for destruction; it’s trying to protect and enhance the function of beneficial proteins that help the body fight disease. Differentiator: Entact’s Encompass platform uses deubiquitinases, or DUBs—enzymes that regulate proteins in cells. Enhancement-targeting chimeric (ENTAC) molecules pair beneficial target proteins with DUBs, which then shorten or remove chains of ubiquitin attached to the target protein, ostensibly enhancing its function and restoring the cell to health. One More Thing: CEO Victoria Richon was formerly founding president and CEO of Ribon Therapeutics, a clinical-stage biotech targeting stress support pathways in cancer and inflammation. 29. Rezo Therapeutics Launched: November 2022 Located: San Francisco, CA Series A: $78M Notable: In Rezo’s Series A announcement, co-founder and President Nevan Krogan called the human body a “complicated mosaic of cell and molecular interactions” and said the field has been relying on a “two-dimensional” view to solve disease. Differentiator: So, the company’s Sequence to Systems to Drugs (SSD) platform integrates data from proteomics, genetics, structural biology and chemical biology approaches. Together, these methods enable the rapid identification and study of disease-causing protein and genetic interactions, according to the announcement. One More Thing: Co-founder and UCSF professor Kevan Shokat developed a successful approach to drugging a protein produced by the mutated KRAS gene, an achievement for which he was honored with two prestigious awards in 2023. 30. Aether Bio Launched: August 2023 Located: Menlo Park, CA Series A: $49M Notable: One of the more unique companies on this list, Aether Bio is using generative AI and automation to find novel biocatalysts. With its molecular assembler platform, Aether intends to create enhanced products across the healthcare, home electronics and automotive industries. Differentiator: Aether’s platform combines several buzzy modalities: high-throughput robotics, machine learning and synthetic biology, with the intention of mapping millions of enzyme-reaction combinations. One More Thing: Aether’s Series A round was led by Jay Zaveri at Natural Capital and Trevor Zimmerman at Unless, an impact investment and VC firm “catalyzing the new industrial revolution.” Correction (Jan. 3): This story has been updated to correctly state that Alterome is based in San Diego not the Bay Area. BioSpace regrets the error.

▎药明康德内容团队编辑在生物医药产业的发展地图上，美国以其独特的商业生态系统，已在旧金山、波士顿、华盛顿州、北卡罗来纳研究园和圣地亚哥五个地区构建了较为完善的产业集群，为生物医药领域提供了可借鉴的经验与案例。此前，创鉴汇已盘点了今年获融资的38家旧金山湾区创新药公司，本文将继续走进另一个引领生物医药发展的重要城市——波士顿。今年，美国波士顿成功举办生物技术创新组织（BIO）2023年度大会，超过20,000人的参会人数验证了该城市在生物医药领域的影响力和凝聚力。自上世纪70年代以来，波士顿在生命科学研究、实验室设施建设，以及创新药物的开发和商业合作等多个方面都展现出了强大的、持续的发展动力，吸引了大批跨国药企和新锐在此设立据点。据不完全统计，2023年至今，美国波士顿共有22家生物医药公司获融资，已披露融资总金额近9亿美元，值得关注的亮点包括：神经系统疾病最受关注：至少4家获融资公司重点布局神经系统疾病药物研发，包括本年度波士顿生物医药融资事件金额排名前三的Rapport Therapeutics和Nido Biosciences。AI药物热度不减：6家获融资公司致力于运用AI平台技术推动新药研发，包括搭建了黑暗基因组数据科学平台的ROME Therapeutics，开发了端到端生成式机器学习和合成生物学平台的JURA Bio，以及构建了AI药物发现引擎的Odyssey Therapeutics等。大额B轮融资频现：共发生6起B轮融资事件，其中4起为超5000万美元的大额融资。第三季度出现融资高峰：共发生7起融资事件，已披露金额约3.2亿美元，其中包括波士顿生物医药领域2023年潜在最高融资额事件，即Rapport Therapeutics完成的1.5亿美元B轮融资。#   Rapport：半年内融资两轮的小分子药物新锐8月23日，Rapport Therapeutics完成了1.5亿美元的B轮融资，该轮融资由Cormorant Asset Management公司领投。值得一提的是，该公司今年已完成两轮融资，除本轮之外，还于3月完成了1亿美元的A轮融资。Rapport Therapeutics成立于2022年，总部位于美国波士顿，是一家临床阶段的生物技术公司，专注于发现和开发神经疾病的精准疗法。该公司的创新药物开发平台聚焦于发现受体相关蛋白（RAP），开发精准靶向这些蛋白的小分子药物，具有革新神经疾病治疗的潜力。据新闻稿介绍，其主要候选药物正在1期临床试验中进行测试，用于治疗耐药性癫痫。Rapport Therapeutics的创始人兼首席科学官David Bredt博士是受体相关蛋白（RAP）生物和治疗应用的开拓者，他在20多年前就发现了RAP及其在调节受体表达和功能中的作用。此外，他还提出利用RAP来靶向特定神经元回路的想法。基于这一理念，杨森公司（Janssen）和Third Rock Ventures于2022年底合作创建了Rapport公司。#   Odyssey：获评2023“新一代生物新锐公司”12月5日，Odyssey Therapeutics宣布完成1.01亿美元C轮融资，所得资金将用于推进其精准免疫调节剂和肿瘤药物的研发。今年，该公司被知名行业媒体BioSpace评选为2023年度新一代生物新锐公司（NextGen Bio "Class of 2023"）。Odyssey Therapeutics成立于2018年，是一家专注于发现、开发和商业化下一代免疫调节剂和肿瘤药物的生物技术公司。2022年1月，Odyssey收购了量子机器学习公司Rahko，为Odyssey的发现平台增加了基于物理的分子模拟、机器学习和计算能力，以实现一流的先导化合物识别和候选药物优化。目前，Odyssey正在构建一个将人工智能和机器学习相结合用于分子设计的发现引擎，一个包含针对多种氨基酸、分子胶和天然产物的专有共价库的化学平台，以及用于新靶点发现的功能基因组学平台。此外，Odyssey正在迅速推进免疫学和肿瘤学项目，专注于那些已被验证但尚无上市的解决方案的靶点。目前的管线包括10个小分子或蛋白质药物项目。#   Nido：获超1亿美元支持的CNS新药公司5月16日，Nido Biosciences宣布完成8660万美元B轮融资，至此，该公司累计融资额达到1.09亿美元。Nido Biosciences成立于2018年，总部位于美国波士顿，致力于利用对人类遗传学和疾病的深入理解，开发创新小分子疗法治疗神经系统疾病。该公司基于人类细胞系的功能基因组学发现平台，通过“量身定制“的筛选手段来发现多种神经肌肉和神经退行性疾病的创新靶点，最初将聚焦于肌萎缩侧索硬化（ALS）和额颞叶痴呆（FTD）。Nido公司的主要候选产品NIDO-361是一种创新小分子，它能够与雄激素受体上的一个独特部位结合，并纠正转录调控紊乱，以恢复健康的细胞功能，正在开发用于治疗脊髓延髓肌萎缩症（SBMA）患者。#   ROME：多家大型药企看好的基因组疗法公司9月12日，ROME Therapeutics宣布完成7200万美元的超额B轮扩展融资，使B轮融资总额达到1.49亿美元。本轮融资的投资者包括强生创新（JJDC）、百时美施贵宝（BMS）、ARCH Ventures、赛诺菲风投（Sanofi Ventures）等。融资所得资金将用于推进其主要候选药物LINE-1逆转录酶抑制剂的早期临床试验，包括评估其安全性和确定最佳剂量的1期研究，以及旨在证明机理的其他研究，拟用于治疗LINE-1表达异常的多种严重自身免疫性疾病（如狼疮等）。LINE-1逆转录酶在患病细胞中表达，而在健康细胞中不表达，因此LINE-1逆转录酶抑制剂可以阻断致病性炎症，而不影响机体对感染的反应。ROME Therapeutics成立于2020年，总部位于美国波士顿，致力于开发治疗一系列严重疾病（包括自身免疫性疾病、癌症和神经退行性病变）的新型疗法，其方法是通过阐明黑暗基因组在人类健康和疾病中的作用——黑暗基因组指的是传统基因组研究范畴之外的广阔基因组，包括病毒样重复元件和非编码序列。ROME公司计划继续推进其早期产品管线及其专有的数据科学平台，该平台使得研究人员能够识别功能活跃的重复元件，并评估它们在疾病中的作用。#   Cerevance：2023“生物技术猛公司”得主2月14日，Cerevance宣布扩大了其B轮融资，再获5100万美元，使其B轮融资的总额达到1.16亿美元。所得资金将用于推进基于其专有的NETSseq平台开发的潜在“first-in-class”项目，包括即将进行的针对帕金森病、肌萎缩侧索硬化（ALS）和精神分裂症的临床试验。今年，Cerevance登上业界知名的生物技术猛公司（Fierce Biotech’s 2023 Fierce 15）榜单。Cerevance成立于2016年，其NETSseq技术平台通过使用抗体和RNA探针对人类大脑组织进行染色，能够发现在疾病大脑组织中过度表达或者过低表达的创新靶点蛋白。该公司的主要候选产品CVN424是一种潜在“first-in-class”、具脑渗透性的非多巴胺能口服GPR6反向激动剂，拟用于治疗帕金森病。在研发合作方面，Cerevance已与默沙东（MSD）达成多年战略研发合作，将利用前者专有的NETSseq技术平台，发现治疗阿尔茨海默病的创新靶点。此外，Cerevance还与武田（Takeda）进行了研究合作，通过识别中枢神经系统中表达的新型靶蛋白以开发针对某些胃肠道疾病的新疗法。#   Ampersand：Flagship孵化可编程药物公司3月7日，Ampersand Biomedicines首次公开亮相，并获得由Flagship投资的5000万美元资金。Ampersand Biomedicines成立于2020年，总部位于美国波士顿，致力于通过创建和编程更有效的治疗方法，研发仅作用于疾病发生部位的更安全、更耐受、更有效的可编程药物，开发并利用平台性技术，解决关键的药物开发挑战，即精准靶向和降低“组织外”毒性。Ampersand Biomedicines已搭建起AND（Address, Navigate, Design）平台，能够对来自整个人体的RNA转录数据进行计算分析，从而构建“人体地图”。基于此平台技术，Ampersand公司拟开发“AND-Body疗法”，旨在打造下一代模块化药物，这些药物能够在身体需要的地方精确工作，而不作用于其他地方。据新闻稿介绍，该药物平台具备推广至“几乎所有疾病”的潜力。读者们请星标⭐创鉴汇，第一时间收到推送免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文由药明康德内容团队根据公开资料整理编辑，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转发/复制至其他平台。转发授权请在「创鉴汇」微信公众号留言联系我们。更多数据内容推荐点击“在看”，分享创鉴汇健康新动态