The Boston region in the U.S. state of Massachusetts (MA) hosts one of the leading biotech hubs in the world , ranking second just in front of the BioHealth Capital Region . Here are twelve of Boston’s most innovative biotech companies. The Boston region has sprouted many influential public biotech companies. One of the most successful in recent years was Moderna, a giant messenger RNA (mRNA) specialist incubated by the VC firm Flagship Pioneering. Moderna would go on to produce one of the first COVID-19 vaccines in 2020. Another prestigious name, Vertex Pharmaceuticals, one of the 14 biotechs with a market capitalization above $100 billion, is also headquartered in Boston. The Boston hub shows few signs of slowing down, and there is a host of biotech companies generating excitement among investors. Table of contents Akouos Akouos Akouos, founded in 2016 focuses on developing gene therapies for hearing loss. The Boston-based biotech company uses adeno-associated viral (AAV) vector-based gene therapy techniques aimed at restoring and preserving hearing across a variety of conditions, from genetic mutations to aging-related hearing loss. In December 2022, Akouos was acquired by Eli Lilly for $487 million, signaling a strong endorsement of its potential within the gene therapy space for hearing loss treatments. This acquisition is part of a broader trend of interest in genetic and cell therapies as a frontier for treating previously intractable conditions. Akouos’s primary therapeutic candidate is AK-OTOF, a gene therapy targeting hearing loss caused by mutations in the otoferlin gene (OTOF), which is crucial for effective hearing. This approach reflects a significant shift towards targeting the genetic underpinnings of hearing loss, a common yet challenging condition to manage effectively with current treatments. The company has raised $162.5 million through funding rounds to support its research and development activities with a $105 million series B round. Cellarity Cellarity Cellarity, a biotech company established by Flagship Pioneering in 2017 and headquartered in Somerville just outside of Boston, represents a shift in drug discovery methods by focusing on cellular behaviors rather than individual molecular targets. This approach aims to better understand and treat diseases by exploring the complexities of cellular functions using computational modeling and machine learning. The company’s funding milestones include a series B round in 2021, where it raised $123 million, followed by a series C round in 2022 that brought in an additional $121 million. Cellarity’s strategy involves integrating high-resolution data and single-cell technologies with computational analysis to identify cellular disease drivers and design medicines aimed at these comprehensive cellular signatures. This technique is poised to offer insights into diseases without known druggable targets and to foster the development of new therapies across various conditions. In January 2024, Cellarity announced a collaboration with Novo Nordisk and Omega. Omega will develop an epigenomic controller for obesity management, while Cellarity will focus on developing a small molecule therapy for metabolic dysfunction-associated steatohepatitis (MASH). This collaboration is part of a broader agreement with Flagship Pioneering to leverage its bioplatform companies for novel cardiometabolic disease treatments. ElevateBio ElevateBio The biotech company ElevateBio was founded in 2017 in Waltham, a city close to Boston. The gene and cell therapy developer has consistently bagged outsized funding rounds, topped by a jaw-dropping $525 million series C round in March 2021. It was only the beginning for ElevateBio, as the company raised $401 million in its series D financing round in 2023. ElevateBio specializes in technology that supports the development of advanced therapies, such as gene editing, induced pluripotent stem cells, and protein, viral, and cellular engineering. Portfolio companies under its wing include AlloVir, which develops T cell therapies against viral infections, the gene editing biotech Life Edit, and the cell therapy player HighPass. Central to ElevateBio’s business model is BaseCamp, a program that provides research and manufacturing services to other companies. ElevateBio has ongoing partnerships to develop and manufacture novel treatments with partners including the institutions Massachusetts General Hospital and Boston Children’s Hospital. Emulate Emulate Emulate was established in 2014, with roots in research conducted at the Wyss Institute for Biologically Inspired Engineering at Harvard University. The Boston-based biotech company focuses on developing “Organ-on-a-Chip” technology, a platform that mimics human organ functions on micro-engineered chips, aiming to improve drug testing and reduce reliance on animal models. Since its founding, Emulate has progressed through several financing rounds, starting with a $12 million series A round in 2014, and culminating in an $82 million series E round in 2021. Since its launch, the company has raised approximately $225 million. Emulate’s technology involves using small, flexible chips that simulate the environment and mechanical forces of human organs. This allows researchers to study the effects of drugs and diseases on human biology more accurately and humanely, potentially speeding up drug discovery and development processes. Emulate has collaborations with key players in the industry, notably with Roche, Takeda, AstraZeneca, and Janssen, to apply its Organ-on-a-Chip technology more broadly in drug discovery. These collaborations aim to create models that are more predictive of human responses, moving towards more personalized medicine approaches. The technology’s potential to refine the preclinical phase of drug development could lead to safer, more effective treatments reaching the market more efficiently. Life Biosciences Life Biosciences Life Biosciences is focused on developing therapeutics that target the biology of aging. The company operates on the premise that aging is not merely a result of random wear and tear but is driven by specific biological mechanisms that can be therapeutically targeted. In its pursuit to address aging-related diseases, Life Biosciences completed a series C financing round in January 2022, raising $82 million. This funding round brought the total raised by the company to over $158 million. The capital is intended to accelerate the development of its innovative therapies across three primary platforms, which focus on mitochondrial uncoupling, chaperone-mediated autophagy (CMA), and epigenetic reprogramming. These platforms aim to develop treatments for a wide range of aging-related conditions, including obesity, NASH, neurodegenerative diseases, and others by targeting fundamental biological mechanisms contributing to aging. Life Biosciences is also involved in partnerships and collaborations to advance the development of its therapies. In May 2023, Life Biosciences announced a cGMP manufacturing partnership with Forge Biologics, aimed at advancing the development of novel gene therapies for aging-related diseases. This is part of the company’s broader efforts to bring groundbreaking therapies to the market. MOMA Therapeutics MOMA Therapeutics MOMA Therapeutics, founded in 2020, focuses on developing precision medicines by targeting the complex mechanisms of molecular machines involved in diseases. In 2022, the Boston-based biotech company secured $150 million in series B funding, intended to support the progression of MOMA’s oncology programs toward clinical trials. MOMA’s drug discovery strategy is based on exploiting the dynamic changes in molecular machine conformation, which are crucial for their enzymatic activities. By focusing on these proteins, MoMa aims to develop medicines that can precisely target and modulate disease-causing proteins, offering new treatments for patients with significant unmet medical needs. In 2024, MOMA initiated a significant collaboration with Roche to discover new cancer treatments. This partnership, valued at $66 million upfront with the potential for over $2 billion in milestone payments and royalties, leverages MoMa’s KnowledgeBase platform to identify novel cancer targets. Neumora Therapeutics Neumora Therapeutics Many neuropsychiatric and neurodegenerative diseases are notoriously difficult to treat, such as schizophrenia and Alzheimer’s disease. Across the decades, many clinical trials have struggled to provide a one-size-fits-all approach that works for many patients. Neumora Therapeutics is exploring a different strategy: precision medicine. Cancer used to be classified and treated according to symptoms and organs affected, but the efficacy of these treatments was lacking, said Paul Berns, co-founder, chairman, and chief executive officer (CEO) of Neumora, in a public statement. Precision medicine made many cancer treatments more targeted and more effective. “Similarly, we now have the tools and technologies to redefine brain diseases to transform the development of targeted, effective precision medicines,” he added. Neumora Therapeutics closed a huge series A round worth $400 million when it launched in October 2021, and bagged another $100 million investment from Amgen. The company raised an additional $112 million in its series B round in 2022. In September 2023, Neumora announced the pricing of its Initial Public Offering (IPO) at $250 million. The Boston-based biotech company is developing Data Biopsy Signatures: maps of different drivers of brain diseases from which it can identify specific types of patients that would benefit from targeted therapy and specially designed clinical trials. These maps are derived from a range of data sources including genomics, imaging, brain electrical activity, and clinical records. Neumora’s lead candidate treatment, Navacaprant, is in phase 3 testing for the treatment of depression. The company is running other programs for the treatment of anxiety and neurodegenerative diseases. Orna Therapeutics Orna Therapeutics Orna Therapeutics was founded with a focus on developing a new class of fully engineered circular RNA therapies, known as oRNA. This approach to RNA therapeutics is built on the concept of transforming linear RNA into a circular form, which presents several advantages over traditional linear mRNA therapies, including simplified production, increased protein expression, and a superior immunogenicity profile. One of the most notable achievements in its financing history was the closing of a $221 million series B financing round in August 2022. This funding was aimed at advancing the company’s circular RNA + LNP (lipid nanoparticle) delivery platform and its lead in situ CAR (isCAR) program toward clinical trials. The round saw contributions from key investors, including Merck. By engineering circular RNAs that self-circularize, Orna’s platform aims to overcome the limitations of existing RNA therapies, offering the potential for more effective and durable treatments. The company’s preclinical data have shown promising results, especially in cancer therapy, where oRNA-based therapies have demonstrated tumor suppression and eradication in animal models. Looking forward, Orna anticipates moving its lead isCAR program into clinical trials in 2024. Sail Biosciences Sail Biosciences Sail Biosciences is the result of the merger between Laronde and Senda Biosciences. When we first started this list in 2022 Laronde was a part of it, however, a lot has changed since then. Sail Biosciences is headquartered in the greater suburb area of Boston, in Cambridge Massachusetts. Laronde was unveiled by Flagship Pioneering in Cambridge, MA, in early 2021. By August 2021, Laronde had bagged $440 million in a series B round to bankroll the development of drugs based on technology called Endless RNA. Drugs based on mRNA like Moderna’s COVID-19 vaccine work by instructing cells to produce therapeutic proteins. However, these drugs can only exert their effects for a short time before being cleared from the body. Endless RNA is designed to overcome this limitation by making RNA molecules into closed loops that last longer in the body. These modular molecules can also be tweaked easily to carry different instructions for making multiple proteins. However, the biotech company’s strong start was soon overshadowed by a Boston Glove investigation pointing at “bad assay” and poor note-taking in the core research of the company resulting in the company having to shelve several candidates. In October 2023 Laronde and Senda biosciences, another RNA company that has raised $226 million since its creation in 2016, announced their merger into Sail Bioscience. The company will combine Senda’s natural nanoparticle platform and Laronde’s promising eRNA, a second chance for this technology. Tessera Therapeutics Tessera Therapeutics Tessera Therapeutics hit the headlines in April 2022 with an impressive $300 million series C financing. The biotech company was founded by Flagship Pioneering in Somerville, close to Boston. The proceeds of its latest round are bankrolling the development of Tessera’s Gene Writing technology, which can make a range of edits and insertions into the genome to deliver therapeutic genes. While lots of hype is on the therapeutic potential of CRISPR technology, Tessera’s Gene Writing technology is based on a different concept called mobile genetic elements (MGEs): a vast group of genomic sequences including plasmids and transposons that are able to move within a genome or even jump to other organisms. The technology can use RNA templates to make small edits to genetic code or DNA templates to insert whole sequences. According to the company, this has the potential to cure nearly any genetic disease, even those that are inaccessible to current gene editing technology. One of the diseases this technology could tackle is cystic fibrosis, a condition caused by a range of genetic mutations in a gene called CFTR . Right now, some treatments can alleviate the condition in patients with certain types of mutations, but some patients with other mutations are left without any options. In 2021, Tessera joined forces with the Cystic Fibrosis Foundation to speed up the development of ways to rewrite the faulty CFTR gene and potentially cure the condition in all patients. Wave Life Sciences Wave Life Sciences Wave Life Sciences is a biotechnology company focusing on the development of RNA-based therapeutics for various diseases. The company leverages its proprietary PRISM platform, a versatile tool for developing RNA medicines, combining multiple modalities and innovative chemistry to address both rare and prevalent disorders. This platform enables Wave to engage with mRNA inside human cells to repair, restore, or reduce proteins to treat diseases, offering advantages over traditional therapeutic approaches, such as avoiding permanent off-target genetic changes and accessing a variety of tissue types. Financially, the Boston-based biotech company has raised $18 million in a series A financing round followed by a $66 series B round. Since its IPO in 2015, the company has raised an additional $583 million in post-IPO rounds. Wave Life Sciences has been active in advancing its research and development pipeline, including programs targeting Huntington’s disease, Duchenne muscular dystrophy, and Alpha-1 antitrypsin deficiency. The company reported progress in these areas, with several programs moving forward in clinical trials. For instance, they are on track to bring WVE-006, the industry’s first RNA editing compound, into clinical trials for Alpha-1 antitrypsin deficiency. The company’s collaborations have been instrumental in expanding its research capabilities and financial resources. One notable partnership is with GSK, aimed at advancing oligonucleotide therapeutics focusing on novel genetic targets. Xtalpi Xtalpi Xtalpi was set up by a team of quantum physicists at MIT in 2014 with the aim to speed up the drug discovery process. It uses a range of digital and computational tools such as quantum physics, artificial intelligence (AI), and cloud computing to predict the chemical properties of small molecule drugs at an early stage of development. With offices in Cambridge and Shenzhen, China, Xtalpi has a range of partnerships on the go with companies around the world such as Singleron, PhoreMost, Signet, and more recently with Janssen and Eli Lilly. Just one year after bagging $300 million in a series C round in late 2020, Xtalpi went on to close a series D mega round worth $400 million in late 2021. Some of the services Xtalpi offers to clients include discovering chemical structures that can hit disease targets; finding the optimal profile for a drug candidate to improve its success rate; and validation of its computational predictions with wet lab experiments. Last month XtalPi announced the launch of Ailux, a new division focused on AI-powered biologics discovery. The division aims to enhance biologics drug discovery by combining proprietary AI algorithms and wet lab methods. Boston, an advanced biotech ecosystem The Boston biotech ecosystem is a dynamic and innovative environment, home to almost a thousand biotechnology companies. This ecosystem is bolstered by the presence of prestigious research labs, universities, and colleges in Boston and Cambridge, creating a fertile ground for biotech innovation and growth. With companies contributing in various areas of the industry, from cancer care to AI drug discovery, the Boston ecosystem has grown to become one of the most complete in the industry. Networking groups, events, accelerators, and incubators play a crucial role in fostering connections and development within the Boston biotech scene. Organizations and events such as the ASAP BioPharma Conference, Bio Pharma Networking Group, and the Harvard Biotech Club provide platforms for professionals to meet, exchange ideas, and collaborate. Additionally, Boston and Cambridge are also home to world-leading U.S. venture capital (VC) firms investing in promising life sciences research. Examples include Third Rock Ventures and RA Capital Management. This article was originally published in July 2022 by Jonathan Smith and has since been updated by Jules Adam on March 2024. Partnering 2030: The Biotech Perspective 2023
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