As 2024 unfolds, the biotechnology industry is brimming with potential, spearheading scientific innovation. This year is particularly exciting as a diverse range of biotech companies are poised to break new ground to tackle global challenges. Here are nine biotech companies we will follow in 2024.
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Aiolos Bio
Aiolos Bio has emerged as a significant player in the biopharmaceutical industry, with a focus on developing treatments for respiratory and inflammatory diseases. The company’s launch in 2023, backed by a remarkable $245 million series A funding round, marked a noteworthy entry into the clinical-stage biopharmaceutical sector.
At the heart of Aiolos Bio’s mission is AIO-001, a humanized anti-TSLP monoclonal antibody, developed as a treatment for moderate-to-severe asthma. This antibody targets thymic stromal lymphopoietin (TSLP), a key cytokine involved in inflammation, which is central to asthma treatment.
AIO-001 distinguishes itself with its high potency and extended half-life, as demonstrated in phase 1 studies. Its potential for biannual administration offers a significant improvement over current treatments like Tezspire, which require monthly injections. This frequency advantage could position Aiolos Bio advantageously in the market, offering patients a more convenient treatment.
Aiolos Bio’s team brings extensive experience in respiratory and immunological diseases. This experience, including the team’s involvement in more than 30 FDA approvals, forms a solid foundation for the company’s ambitious objectives.
The company’s acquisition of global development and commercialization rights for AIO-001 from Jiangsu Hengrui Pharmaceuticals was a strategic move that broadened its international presence.
In 2024, Aiolos Bio is expected to make pivotal advancements. The planned phase 2 clinical trial for AIO-001 in moderate-to-severe asthma patients will be a key event. The company’s dedication to addressing unmet needs in asthma and other immune disorders reflects its commitment to patient-centric innovation.
Aiolos Bio’s methodological approach to treatment, focusing on efficacy and patient convenience, coupled with robust funding and experienced leadership, positions it as a biotech company to observe closely in 2024. Its progress in the development of AIO-001 and potential expansion into other respiratory and inflammatory diseases could have a significant impact on patient care in these fields.
Capstan Therapeutics
Based in San Diego, Capstan is dedicated to developing therapies that involve precise engineering of cells within the body. This approach is different from traditional methods that typically modify cells outside the body and then reintroduce them.
The company’s research targets a variety of diseases, including oncology, autoimmune diseases, blood disorders, and fibrosis. Capstan’s technology centers around targeted lipid nanoparticles (tLNPs) for delivering mRNA payloads to cells. This approach aims to enable the in vivo generation of CAR-T cells, a method that could offer a more efficient and potentially less costly alternative to current CAR-T cell therapies.
The technology builds on research from the University of Pennsylvania, combining expertise in mRNA and cell therapy. The company launched with a notable $165 million in financing, a strong statement underlining investors’ confidence in their approach and potential.
Capstan’s work focuses on developing treatments that could be administered in outpatient settings, which is promising for diseases lacking effective treatment options. This could lead to more accessible and potentially less invasive therapies. The company’s work on in vivo generation of CAR-T cells represents a novel approach in the field of immunotherapy. If successful, this could significantly advance the treatment of cancer and potentially other diseases.
Carisma Therapeutics
Carisma Therapeutics, emerging as a trailblazer in the biotech industry, is particularly noteworthy for its innovative approach to cancer treatment. This clinical-stage biopharmaceutical company has been focused on leveraging its proprietary macrophage and monocyte cell engineering platform to develop transformative immunotherapies aimed at treating cancer.
A significant development for Carisma in recent years has been its strategic collaboration with Moderna. This collaboration has brought forward the development of in vivo CAR-M (chimeric antigen receptor macrophage) therapeutics, a novel and promising approach in the field of oncology. In 2023, Carisma announced the nomination of its first lead candidate under this collaboration, marking a significant milestone in the development of CAR-M therapies.
The lead candidate developed by Carisma, in collaboration with Moderna, targets antigens present on solid tumors, addressing a critical area of unmet medical need in cancer treatment. The pre-clinical proof of concept data, showcased at SITC 2023, underlined the feasibility, efficacy, and tolerability of this innovative approach. This data suggests that Carisma’s CAR-M therapy, produced directly in vivo, can effectively redirect endogenous myeloid cells against tumor-associated antigens.
Carisma’s CAR-M therapies represent a significant shift in cancer immunotherapy. They offer an off-the-shelf solution, potentially increasing access to CAR-based therapies and laying the groundwork for future CAR-M programs under the Carisma and Moderna collaboration. The potential of these therapies to revolutionize cancer treatment, especially in the context of solid tumors, positions Carisma as a biotech company worth following closely in 2024.
Cellarity
Cellarity was launched by Flagship Pioneering in 2017. Unlike traditional methods focusing on single molecular targets, Cellarity’s approach is to understand and rectify cellular dysfunction, offering a more holistic view of disease biology. The company has developed a robust platform utilizing proprietary AI models trained on over 30 million single-cell transcriptomes. This innovative method allows Cellarity to uncover novel biological insights and create unique drug candidates across a range of diseases, including metabolic disease, hematology, and immuno-oncology.
In 2022, the company raised $121 million in series C funding, bringing its total funding to $274 million. This significant investment is a strong indicator of investor confidence and the promise of Cellarity’s drug discovery strategy. The approach of observing cellular transitions from health to disease and leveraging machine learning to reverse engineer this process could lead to a better understanding of complex diseases.
In 2023, Cellarity was nominated for the Prix Galien USA Award in the Best Startup category. More recently, Cellarity has announced its partnership with the Chan Zuckerberg Initiative. This collaboration aims to advance machine learning algorithms for single-cell analysis, addressing challenges in modeling cellular response to chemical perturbations. we will be watching this biotech company’s evolution closely in the course of 2024.
Kriya Therapeutics
Kriya Therapeutics is shaping up to be a significant company in the biotech industry for 2024, demonstrating a robust approach to gene therapy with impressive financial backing ($600 million, according to Crunchbase) and strategic advancements. Kriya’s product pipeline addresses diseases of high unmet need across various therapeutic areas, including ophthalmology, oncology, rare diseases, and chronic diseases. Their approach involves targeting biological pathways with gene therapy to deliver transformative benefits.
Kriya’s acquisition of Warden Bio, a company developing novel AAV-mediated gene therapies for glycogen storage disorders (GSDs), marked a significant expansion of their pipeline. This acquisition underpins Kriya’s Rare Disease Division, emphasizing its potential to discover and develop gene therapies for rare diseases. Such strategic movements are essential for Kriya’s growth and diversification in the field.
Kriya Therapeutics has successfully secured substantial financial support, a notable $270 million was raised in their series C financing, led by Patient Square Capital. This funding is intended to advance Kriya’s pipeline and support the scaling of its engineering, manufacturing, and computational platforms. This financing brought Kriya’s two-year total to $450 million, solidifying its financial foundation to build out its all-in-one gene therapy business.
Kriya has established a state-of-the-art, scalable GMP manufacturing infrastructure in Research Triangle Park, North Carolina. This facility supports in-house production from early through late-phase development. Additionally, the company has developed a computational machine learning platform, SIRVE, to efficiently develop and test new gene therapies. The focus on building robust infrastructure reflects Kriya’s commitment to advancing the field of gene therapy.
Maze Therapeutics
Maze Therapeutics, a South San Francisco-based biopharmaceutical company, operates at the intersection of human genetics and drug development. The company’s primary mission is to translate complex genetic insights into a new generation of precision medicines. Maze’s approach is particularly notable for its emphasis on understanding genetic drivers of common diseases, an area that has been traditionally challenging in the field of drug discovery.
The company’s research spans a broad spectrum of conditions, with a significant focus on diseases such as kidney disorders, which are often characterized by a lack of effective treatment options. This focus is exemplified by their development of MZE829, a novel treatment candidate for APOL1 kidney disease, a condition prevalent in specific populations and linked to genetic factors.
One of Maze’s prominent focuses is APOL1 kidney disease. The company has initiated a phase 1 trial for MZE829, an oral APOL1 inhibitor, marking a pivotal step in its journey to provide disease-modifying treatments. This initiative demonstrates Maze’s potential to offer innovative solutions for conditions with limited treatment options.
Sanofi had entered a licensing deal with Maze for the Pompe disease program MZE001. This deal, announced in May 2023, involved Sanofi paying $130 million upfront, plus up to $605 million in milestones, for worldwide rights to a drug candidate aimed at treating Pompe disease by stopping the buildup of glycogen.
However, this deal was scrutinized by the Federal Trade Commission (FTC), which blocked the agreement citing antitrust concerns. The FTC’s complaint alleged that the deal would have effectively eliminated competition by buying out Maze rather than competing with it. In response, Sanofi decided to back off from the deal instead of contesting the decision.
2024 will be an interesting year for this biotech company, between their response to this development regarding MZE001 as well as the phase 1 trial for MZE829.
Quotient
Quotient Therapeutics, a recent venture of Flagship Pioneering, has garnered attention in the biotechnology industry with its innovative approach to somatic genomics. Based in both Cambridge, MA, and Cambridge, U.K., Quotient Therapeutics was launched with a significant $50 million investment from Flagship Pioneering. This biotech company is definitly worth looking at in 2024.
Central to Quotient’s innovative approach is its advanced genome sequencing technology, Nanoseq, licensed from the Wellcome Sanger Institute. This technology allows for the identification of genetic variations at an unprecedented cellular level. Utilizing laser microdissection, the technology can isolate and identify cells or small groups of cells that have accumulated cancer-driver mutations. This method provides a groundbreaking way to study natural selection at the cellular level and offers a window into the vast genetic variation.
Quotient’s Somatic Genomics platform leverages this technology to uncover extensive variations encoded in the somatic genome. The platform’s process includes phenotyping of cells from clinical tissue samples, isolation, single-cell genotyping, and computation. Quotient Therapeutics is well-positioned to make significant contributions to the field of biotechnology. The company’s focus on the vast genetic variation within individuals opens up new possibilities for personalized medicine and the development of transformative therapies.
ReNAgade Therapeutics
ReNAgade Therapeutics, focussing on developing RNA Medicines, has made a significant entry into the industry. ReNAgade launched with a $300 million series A financing round, one of the largest of its kind in 2023. This substantial backing is a strong indicator of investor confidence in the company’s mission and potential.
The company aims to harness the potential of RNA medicines to treat diseases across various parts of the body. Leveraging novel RNA delivery platforms, ReNAgade combines a comprehensive RNA platform for coding, editing, and gene insertion, aiming to develop new, transformative medicines. This approach puts them at the forefront of RNA-based therapeutic development, seeking to expand the potential of RNA medicines beyond current limitations.
ReNAgade’s technology focuses on a broad portfolio of proprietary RNA delivery systems, including novel lipid nanoparticles (LNPs). These systems, combined with genomic coding, editing, and insertion technologies, are designed to precisely correct disease on an unprecedented scale. By exploring the full range of RNA medicine capabilities, ReNAgade aims to develop treatments that can address a wide array of diseases, potentially leading to groundbreaking advancements in medical science.
ReNAgade has established a Joint Venture with Orna Therapeutics, combining its delivery platform with Orna’s circular RNA technology.
Looking ahead to 2024, ReNAgade Therapeutics is poised to make substantial strides in the field of RNA medicine. The integration of cutting-edge technology, combined with a comprehensive approach to RNA medicine, places ReNAgade as a biotech company to watch in 2024 and in the coming years.
OrsoBio
OrsoBio is pioneering the development of innovative therapies for severe metabolic disorders. The company’s focus encompasses a broad spectrum of conditions, including type 2 diabetes, severe dyslipidemias, lipodystrophies, and nonalcoholic steatohepatitis (NASH). OrsoBio’s approach is characterized by a commitment to addressing central pathways in energy metabolism, potentially revolutionizing the treatment landscape for these challenging conditions.
The company’s recent $60 million series A financing, bringing the total capital raised to $97 million, marks a crucial phase in its journey. This funding is expected to support three phase 2 studies through the end of 2024, advancing OrsoBio’s mission to redefine the treatment of severe metabolic disorders.
OrsoBio’s portfolio includes four key programs in clinical and preclinical development:
- Mitochondrial Protonophores: TLC-6740 and TLC-1235, targeting severe lipodystrophies and other metabolic disorders, are key assets in OrsoBio’s arsenal. These protonophores are designed to increase energy expenditure in patients, thus addressing fundamental aspects of metabolic disorders.
- ACC2 Inhibitor Program: TLC-3595, an oral acetyl-CoA carboxylase-2 (ACC2) inhibitor, is another promising candidate. ACC2 inhibitors are being explored for their potential to improve mitochondrial function in patients with metabolic and inflammatory liver and kidney disorders. Early trial results for TLC-3595 have shown it to be well tolerated, with positive impacts on LDL and total cholesterol levels.
- Human Liver Organoid (HLO) Platform: OrsoBio is leveraging a novel HLO platform, innovated by Dr. Takanori Takebe, to optimize the development of its compounds. This platform is expected to enhance the understanding of disease biology and the therapeutic potential of OrsoBio’s candidates.
- The company is also focusing on aminocarboxymuconate semialdehyde decarboxylase (ACMSD) inhibitors, aimed at treating metabolic and inflammatory liver and kidney disorders. These inhibitors could significantly advance the treatment of diseases characterized by impaired fatty acid oxidation (FAO).
OrsoBio’s innovative approach, backed by significant funding, positions it as a prominent player in the biotech industry. The company’s focus on developing therapies for severe metabolic disorders makes it a key biotech company to watch in 2024. As OrsoBio moves forward, its advancements could offer new hope for patients suffering from these challenging conditions.
