Follow the Money: Lipid Nanoparticle Delivery Platform, Regulatory RNA Targeting, More

$407M: Refinancing Deal for Pylera Commercial Rights 

The French specialty pharmaceutical company, Juvisé Pharmaceuticals, completed a transaction with AbbVie to acquire the worldwide commercial rights of Pylera. Juvisé Pharmaceuticals raised €400 million in financing with Société Générale, the company’s historic and lead bank, and supported the funding with the refinancing of its existing debt. Juvisé Pharmaceuticals has established successful alliances with world-class pharmaceutical actors, per previous transactions with Novartis and AstraZeneca. 

$258M: Funding Awards for New Research Studies

The Patient-Centered Outcomes Research Institute (PCORI) approved funding awards totaling $258 million to support 26 new research studies. These studies aim to fill evidence gaps and improve health care decision-making for mental health, heart disease, substance use disorders, and a range of high-burden conditions, including asthma, bone fractures, migraines, intellectual and developmental disabilities, and sinus infections. Notable awards include $31 million to The American Academy of Family Physicians, in partnership with Penn State College of Medicine, for the study of asthma treatments and $7 million to a Dartmouth-led research group studying the correlation between an older adult patient’s quality of life and various methods of providing office visit information. Details of all studies and projects approved for PCORI funding are available on the organization’s website.

$120M: Series B Extension for Targeted Genetic Therapeutics 

ReCode Therapeutics, a genetic medicines company, announced the close of a Series B extension financing round for a total of $200M. The initial Series B funding was co-led by Pfizer Ventures and EcoR1 Capital. ReCode’s lipid nanoparticle delivery platform enables targeted delivery of genetic therapeutics directly to the organs and cells most impacted by disease, offering improved efficacy and potency. With the additional $120M in new financing, ReCode will diversify its pipeline into CNS diseases, advance lead mRNA programs for primary ciliary dyskinesia and cystic fibrosis, and develop a platform to deliver a broader range of genetic medicine cargoes. 

$100M: Series B Funding for RNA-Targeting Platform 

CAMP4 Therapeutics announced closing a $100 million Series B round to advance the company’s lead regulatory RNA (regRNA) target programs and accelerate regRNA Actuating Platform (RAP) expansion. CAMP4’s approach targets regRNAs, RNA that control the expression of nearby protein-encoding genes. RAP maps regRNAs associated with every protein-coding gene in any cell type to upregulate gene expression to treat disease. This approach applies to various genetic disorders where increases in gene output can lead to meaningful therapeutic outcomes. While the company is initially focusing on the central nervous system and liver conditions, its platform has the potential to address a broad range of genetic indications across multiple tissues, with a focus on haploinsufficient diseases. 

$65M: Contract for Cloud and Data Integration 

General Dynamics Information Technology (GDIT), a business unit of General Dynamics, announced that it was awarded a new $65 million contract by the Centers for Medicare & Medicaid Services (CMS) Enterprise Architecture Data Group to support its Integrated Data Repository (IDR). CMS maintains the largest volume of health care data files in the world. The IDR is a high-volume data warehouse integrating claims, beneficiary, and provider data sources to support various Medicare and Medicaid programs. Access to this robust integrated data supports insights into medical trends, healthcare costs, fraud, waste, and abuse. Under this contract, GDIT will provide agile transformation, development, security, and operations services supporting on-premises and cloud systems. 

$47M: Series A Funding for Cancer Inhibition 

Faeth Therapeutics, a cancer metabolism company, announced closing a $47 million Series A round of private financing, bringing total funding to $67 million. The financing proceeds will advance Faeth’s clinical trials that combine nutrient control, therapeutics, and digital tools to inhibit cancer metabolism. The company has now moved to first-in-human clinical trials looking at pancreatic, colon, endometrial, and ovarian cancers. 

$40M: Series A Funding for Protein Engineering Platform 

Manifold Bio, a next-generation protein therapeutics company pioneering an in vivo biologics design platform, announced the close of a $40 million Series A financing round. The investment will enable the company to advance its internal drug programs and expand its next-generation protein engineering platform to support additional programs and partnerships. Founded by Professor George Church, Ph.D., and leading biological engineers from his laboratory at Harvard Medical School and the Wyss Institute for Biologically Inspired Engineering, Manifold Bio’s mission is to leverage technological innovation to address critical translational bottlenecks in drug creation. 

$36M: Funding for Patient Engagement Research 

The Patient-Centered Outcomes Research Institute (PCORI) launched a three-year initiative offering up to $36 million to strengthen and enhance the evidence base on engagement in research with the posting of an initial Science of Engagement PCORI Funding Announcement (PFA) that offers up to $4.5 million for foundational research. PCORI seeks to fund studies that focus on two foundational areas: developing or validating measures that assess the structure, process, and outcomes of engagement; and the development and testing of engagement techniques to generate evidence on the most effective engagement approaches, particularly for underrepresented populations. Letters of Intent for this PFA are due August 9 by 5 pm (ET), and applications will be due November 15 by 5 pm (ET). 

$19M: Series A Extension for Drug Discovery 

Xilis, a biotechnology company developing its MicroOrganoSphere (MOS) technology to guide precision cancer therapy and accelerate drug discovery and development, closed an extension of over $19 million to its Series A financing round, bringing the total amount raised to over $89 million. The company will use the Series A funding to advance its proprietary MOS platform. This innovative MOS technology is the first genuinely scalable patient-derived model that recapitulates the tumor microenvironment and immune interactions necessary for next-generation immuno-oncology and cell therapy drug discovery. 

$18M: Equity Financing for Science Learning Platform 

ACTO, the omnichannel learning and enablement platform for life sciences, announced closing an $18 million equity financing. The company will use the proceeds to accelerate the growth and scale of ACTO and explore potential business and partnership opportunities. This round of funding allows ACTO to expand and enhance capabilities to accelerate its technology. The current financing brings ACTO's total invested capital to more than $30 million. 

$14M: Funding Round for Genetic Testing Platform 

Nucleus Genomics, a next-generation consumer genetic testing and analysis company, has raised $14 million in a new funding round led by Alexis Ohanian's Seven Seven Six. The NYC-based company is building a direct-to-consumer genetic testing platform that provides innovative genetic risk and disposition score analyses on many diseases and traits by leveraging whole-genome sequencing (WGS). The additional funding will help Nucleus bolster its scientific and engineering team. They continue to set up the computational and physical infrastructure required to analyze WGS data at scale, customize disease and trait reports, and send genetic testing kits. 

$8M: Renewed Funding for Patient Data Network Expansion 

The INSIGHT Clinical Research Network–a Weill Cornell Medicine-led database of more than 15 million patients–received nearly $8 million in renewed funding from the Patient-Centered Outcomes Research Institute to expand the database and its use for health research. The network is a collection of de-identified electronic health records and clinical trials data from the five top academic medical centers in New York City, centralized into one database. The study seeks to leverage patient-driven data to address fundamental scientific questions, such as disease manifestations over time and the susceptibility of specific populations to adverse outcomes. INSIGHT also received a $9.8 million grant from the National Institutes of Health to co-lead a nationwide consortium of more than 40 health care institutions analyzing electronic health record data to detect, predict, treat, and prevent long COVID. 

$5M: Funding for Clinical Data Cleaning Platform 

Cornerstone AI announced a $5 million seed round to launch a first-of-kind artificial intelligence platform. Cornerstone develops intelligent software to efficiently clean and prepare data in a fraction of traditional methods. With Cornerstone AI, a medical device company reduced its six-month manual data cleaning protocol to nine days. Similar results were seen using medical record data, clinical trial/registry data, and digital health data. Funds will be used to further product development and scale the business. 

$4.2M: Funding for Proteogenomics Data Analysis Center 

The Office of Cancer Clinical Proteomics Research at the National Cancer Institute awarded $4.2 million to Mount Sinai researchers to establish a Proteogenomic Data Analysis Center for advancing cancer research and treatments. Proteogenomics, which studies the roles of proteins and genes within a cell or organism, is a crucial area of exploration to further the understanding of cancer. The award supports the Center’s work to identify potential biomarkers and drug targets for cancer, new insights into cancer biology, and bioinformatics tools to enable more advanced exploration and discovery from cancer-related datasets. 

$2.8M: Funding for Pediatric Neurological Symptoms 

PARROT, a new landmark clinical trial, launched in the UK with an additional £2.3m in funding by the National Institute for Health and Care Research (NIHR) for a total of £3.1m. PARROT is the joint UK and Australia clinical trial led by researchers from the University of Liverpool and Alder Hey Children’s NHS Foundation Trust on children with neurological impairment. It aims to recruit 500 children and young people aged 3-17 to determine whether a 12-month treatment of azithromycin affects rates of hospital visits, chest infections, General Practices and Accident and Emergency visits, prescriptions, medicines, and the quality of life of both parent and child. 

$2.6M: Funding for ALS Vaccine 

DZNE and Intravacc B.V., contract development and manufacturing organization of preventive and therapeutic vaccines, were awarded €2.5 million from the European Union to develop an ALS vaccine prototype to be clinically tested in humans. About 5-10% of all ALS cases are caused by a mutation in the C9orf72 gene, making it the most common genetic ALS variant. DZNE's experimental vaccine instructs the immune system to produce antibodies against the variant’s resulting protein.