Medical, Diagnostics
prnewswire | August 28, 2023
Aspen Neuroscience today announced that it is partnering with Emerald Innovations, a pioneer in the creation of "invisible" off-body sensors for measuring health analytics, and Rune Labs, a software and data analytics company for precision neurology, to incorporate digital health (DHT) modalities in the company's Trial-Ready Screening Cohort Study.
The Trial Ready Screening Cohort study was launched in 2022 to screen, enroll and begin manufacturing cells for potential patient candidates for the future Phase 1/2a clinical trial for ANPD001, a personalized (autologous) cell therapy to treat Parkinson's disease (PD) by replacing lost dopamine neurons.
Aspen Neuroscience is advancing the collection of objective measures of motor function via passive in-home monitoring, working with Emerald Innovations' contactless biosensors, and active monitoring, working with Rune Labs' clinical development platform, StriveStudy. The use of DHT empowers trial investigators to include objective, long-term (pre-treatment) motor and sleep symptom capture, to provide data on each patient's rate of disease progression.
"Parkinson's disease is very personal in nature, and we are working to develop a personal cell therapy for PD," said Damien McDevitt, PhD, Aspen president and CEO. "Everyone with Parkinson's has a unique experience, with varied symptoms. We look forward to working closely with the innovative and collaborative teams at Rune Labs and Emerald Innovations to capture a holistic and precise pattern of motor symptom activity over time for people in our screening study."
"Parkinson's is a heterogeneous condition, adding to the challenge of assessing treatment benefit without a personalized view of each trial participant's symptom activity," said Brian Pepin, CEO of Rune Labs. "Through StriveStudy, our biopharma partners are able to collect large quantities of continuous multimodal data to characterize participant's disease activity across a range of clinical features. We are excited to collaborate with Aspen Neuroscience to help them characterize patient symptoms as they bring their personalized cell therapy into clinical testing."
"We are delighted to collaborate with Aspen Neuroscience," said Dr. Dina Katabi, MIT professor and the co-founder and president of Emerald Innovations, Inc. "A significant hurdle in Parkinson's disease clinical trials is the reliable monitoring of disease progression and medication response without the need to enroll hundreds or even thousands of patients. Empirical evidence has demonstrated that readings from the Emerald sensor offer a dependable marker for tracking disease progression. The Emerald sensor gathers this data without body contact, relying solely on the analysis of radio signals in the environment as patients go about their normal lives."
About DHT in the Trial Ready Cohort Screening Study
Aspen will partner with Rune Labs to deeply characterize each patient's symptoms in the Trial Ready Cohort, including the collection of multimodal data, thereby enabling robust disease characterization over time. This will include objective measures of tremor and dyskinesia levels captured on FDA-cleared StrivePD on the Apple Watch, medication monitoring, as well as Apple HealthKit, including global activity (e.g., daily steps).
Aspen and Emerald will collaborate to assess passive "invisible" in-home monitoring of participants enrolled in the Trial Ready Cohort. This will include gait speed measured across walking intervals, sleep onset, the amount of time between entering different sleep stages; sleep duration, sleep efficiency and WASO: the total time spent in the WAKE stage after sleep onset.
About Parkinson's Disease
Parkinson's disease is the world's second most common neurodegenerative disease, affecting more than 10 million people worldwide, including more than 1 million in the U.S., where an additional 90,000+ people are diagnosed every year. Patients can experience non-motor symptoms such as mental and behavioral changes, sleep problems, depression, memory difficulties and fatigue; and motor symptoms caused by the loss of dopamine neurons in the brain, including unintended or uncontrollable movements (dyskinesia), shaking (tremor), stiffness, and difficulty with balance and coordination, which worsen over time.
By the time of diagnosis, it is common for people to have lost the majority of cells in the brain that make dopamine. To date, there is no disease-modifying therapy that can stop, replace or prevent the loss of dopamine neurons or slow the progression of PD.
About Aspen Neuroscience
Headquartered in San Diego, Aspen Neuroscience, Inc. is a clinical development-stage, private company focused on personalized (autologous) regenerative medicine. The company is developing patient-derived iPSCs to create personalized cell therapies that address diseases with high unmet medical needs, beginning with autologous neuron replacement for Parkinson's disease and extending across the brain and affected organs.
A leading iPSC platform company, Aspen combines stem cell biology with the latest artificial intelligence and genomic approaches to investigate patient-specific, restorative treatments. The company has developed a best-in-class platform to create and optimize iPSC-derived cell therapies, which includes in-house bioinformatics, manufacturing and QC.
About Emerald Innovations, Inc.
Emerald Innovations is a health analytics company that specializes in digital health solutions for passive, contactless in-home monitoring. Emerald seamlessly captures continuous health data from patients as they go about their normal lives. The Emerald biosensors fall under a cutting-edge category known as "Invisibles." Installed in the homes of study participants, these sensors continuously analyze surrounding radio signals. Using artificial intelligence, they extract patients' physiological data while prioritizing their privacy. Emerald simplifies clinical trials for its clients by obtaining statistically significant biomarkers, making them readily available to clinicians and data analysts, and consistently providing updates on the progress of clinical trials. Emerald's clientele encompasses big pharma, Fortune 500 pharmaceutical firms, innovative biotech companies, and leading disease research foundations.
About Rune Labs
Rune Labs is a software and data analytics company for precision neurology, supporting care delivery and therapy development. StrivePD is the company's care delivery ecosystem for Parkinson's disease, enabling patients and clinicians to better manage Parkinson's by providing access to curated dashboards summarizing a range of patient data sources, and by connecting patients to clinical trials. For therapeutics development, biopharma and medical device companies leverage Rune's technology, network of engaged clinicians and patients, and large longitudinal real-world datasets to expedite development programs. The company has received financial backing from leading investors such as Eclipse Ventures, DigiTx, TruVenturo and Moment Ventures. For more information, please visit runelabs.io and strive.group.
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MedTech, Medical
Businesswire | August 21, 2023
BioSkryb Genomics, a company transforming single-cell analysis by enabling the most complete multiomic view of the cellular ecosystem, from discovery to diagnostics, today announced the issuance of a composition of matter patent, US Patent 11,643,682 by the U.S. Patent and Trademark Office related to its novel Primary Template-Directed Amplification (PTA) method and applications for research, diagnostics, and treatment. The patent is exclusively licensed to BioSkryb by St. Jude Children’s Research Hospital.
As science continues to evolve, somatic mosaicism (variants accumulated in DNA after fertilization) has become more broadly accepted as a key influencer of biology and disease, including cancer, cardiovascular disease, and neurological disorders. The challenge in understanding this clonal differentiation and how it influences biological change has been underpinned by the lack of technologies that can provide a precise and holistic view of genomes of individual cells without artifacts or bias.
Primary template-directed amplification was invented to address these challenges by providing a highly accurate, scalable, and efficient nucleic acid amplification and sequencing method for research, diagnostics, and treatment. PTA is the flagship technology that drives BioSkryb’s industry-leading products, including ResolveDNA® and ResolveOME™, a first of its kind multiomic solution that provides whole genome and whole transcriptome analysis from the same single cell in a single workflow.
Since the founding of BioSkryb when the PTA technology was licensed, the company has dramatically improved the chemistry in terms of performance, throughput, and modality with these products, including the ability to utilize the chemistry for whole genome, whole exome, and panels across varying cell types and nuclei. In addition to optimizing the DNA-based chemistry, BioSkryb has further developed the technology by layering on the ability to analyze the corresponding transcriptome and proteome within individual cells allowing researchers and clinicians a complete and integrated view of the microenvironment of a cellular ecosystem, empowering breakthrough discoveries and ultimately improved understanding and treatment of complex diseases.
“This patent serves as not only as a cornerstone of BioSkryb’s IP portfolio, but as the foundation of our best-in-class single-cell analysis products,” said Suresh Pisharody, CEO, BioSkryb Genomics. “PTA is the unique engine that drives our ability to deliver comprehensive integrated multiomics and paves the way for next generation single-cell analysis and research that could lead to better outcomes for patients.”
“The performance of PTA, even in its infancy, was so remarkable that the BioSkryb founding team believed it could serve as the foundation for a company that could transform genomics,” said Jay West, CTO and Co-Founder of BioSkryb Genomics. “Since the filing of this original patent application, we have generated approximately 60 other patents and patent applications in several key geographical markets that have allowed us to build upon the original power of this transformational technology.”
As of August 2023, BioSkryb Genomics, Inc., owns or exclusively licenses one issued U.S. patent and three issued international patents (Europe, Australia, and Hong Kong). There are 58 pending patent applications, including 12 in the United States, 39 international applications, and 7 applications filed under the Patent Cooperation Treaty.
About BioSkryb Genomics
BioSkryb Genomics is a rapidly growing organization that is transforming single cell molecular discovery and analysis. Through its single cell whole genome and whole transcriptome amplification tools, scientists and clinicians can gain an unprecedented view of the genome, transcriptome, and proteome within a single cell to better understand the drivers, mechanisms, and management of complex disease. The company is headquartered in Durham, North Carolina.
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Medical
Businesswire | July 11, 2023
Melinta Therapeutics, LLC a commercial-stage company providing innovative therapies for acute and life-threatening illnesses, announced today a partnership with the Biomedical Advanced Research and Development Authority (BARDA) to advance two antibiotics currently FDA-approved for adults, BAXDELA® (delafloxacin) and VABOMERE® (meropenem and vaborbactam), for use in pediatrics. In addition, Melinta and BARDA will partner on the development of BAXDELA® against biothreat pathogens. Under this contract, BARDA has awarded $20.5M for the base period, with the potential of additional funding of $121.4M, amounting to total funding up to $141.9M if all options are exercised. With this BARDA funding, Melinta aims to submit four supplemental New Drug Applications (sNDAs) for these new indications.
BARDA is part of the U.S. Department of Health and Human Services’ Administration for Strategic Preparedness and Response. This partnership may enable BAXDELA® and VABOMERE® to be integrated into national preparedness efforts as well as pediatric care for multi−drug-resistant bacterial infections, enhancing sustained availability of the products, increasing end-user familiarity, and striving for availability for the US pediatric population.
"I am thrilled and honored that BARDA and Melinta have agreed to partner for this important research marking a significant milestone in our journey toward becoming legendary for providing innovative therapies to people impacted by acute and life-threatening illnesses,” said Christine Miller, Melinta President and CEO. “This contract fuels our excitement to explore expanded applications of BAXDELA® and VABOMERE® through innovative research. We are poised to make a lasting impact in healthcare, unlocking new avenues for patient care and addressing unmet needs. We will drive progress and redefine what is possible, paving the way for a healthier future.”
BAXDELA® (delafloxacin) is a novel fluoroquinolone (FQ), approved for adults in IV and oral tablet formulations to treat acute bacterial skin and skin structure infection (ABSSSI) and community-acquired bacterial pneumonia (CABP). CABP remains a leading cause of pediatric hospitalization and mortality in the US, with the highest incidence and morbidity in children under 2 years of age. The partnership with BARDA will support the BAXDELA® pediatric development plan to extend the adult CABP indication to children aged 2 months to less than 18 years.
In addition, Melinta aims to advance BAXDELA® as a potential treatment option against biothreat pathogens for both adults and children. Given its broad spectrum of activity and availability in both IV and oral formulations, BAXDELA® can provide flexibility for use following a biothreat public health emergency, in varied care settings and across spectrums of disease severity.
VABOMERE® is an approved combination of the carbapenem, meropenem, and the novel ß-lactamase inhibitor (BLI), vaborbactam. VABOMERE® was specifically designed to address gram-negative bacteria that produce beta-lactamase enzymes, including strains producing the Klebsiella pneumoniae carbapenemase (KPC) enzyme, the predominant form of carbapenem-resistant Enterobacterales (CRE) in the US. The CDC (Centers for Disease Control) classifies CRE as an “urgent” threat because there are few alternative antibiotics to treat CRE-infections. The VABOMERE® pediatric development plan will support the filing of sNDAs to extend the adult cUTI indication to children aged 3 months to less than 18 years and target the indication of late onset neonatal sepsis in children aged less than 90 days.
“The ability to partner with BARDA on these important programs is exactly the type of work that brought me to Melinta and what excites me about the work we do,” said Douglas Girgenti, MD, VP, Head of Development and Clinical Operations, Melinta. “Pediatric clinical development of BAXDELA® and VABOMERE® will help inform drug safety and tolerability as well as determine optimal antibiotic dosing, with the ultimate goal of enabling use in pediatric cases and expanding the arsenal of healthcare providers in their fight to save lives. The potential addition of BAXDELA® to the biodefense armamentarium will likewise provide a much-needed option for public health emergencies in both children and adults.”
This project has been funded in whole or in part with federal funds from the Department of Health and Human Services; Administration for Strategic Preparedness and Response; Biomedical Advanced Research and Development Authority, under contract number 75A50123C00022.
About Melinta Therapeutics
Melinta Therapeutics is a biopharmaceutical company dedicated to providing innovative therapies to people impacted by acute and life-threatening illnesses. We focus our expanding portfolio on serving patients with an unmet need because that’s how we make the most meaningful impact. At Melinta, we’re visionaries dedicated to innovation while staying grounded in what matters most: patients. Our portfolio currently includes seven commercial-stage products: BAXDELA® (delafloxacin), KIMYRSA® (oritavancin), MINOCIN® (minocycline) for Injection, ORBACTIV® (oritavancin), REZZAYO™ (rezafungin for injection), TOPROL-XL® (metoprolol succinate) and VABOMERE® (meropenem and vaborbactam).
About BAXDELA® (delafloxacin)
BAXDELA® is indicated in adults for the treatment of acute bacterial skin and skin structure infections (ABSSSI) caused by the following susceptible microorganisms: Staphylococcus aureus (including methicillin-resistant [MRSA] and methicillin-susceptible [MSSA] isolates), Staphylococcus haemolyticus, Staphylococcus lugdunensis, Streptococcus agalactiae, Streptococcus anginosus group (including Streptococcus anginosus, Streptococcus intermedius, and Streptococcus constellatus), Streptococcus pyogenes, and Enterococcus faecalis, Escherichia coli, Enterobacter cloacae, Klebsiella pneumoniae, and Pseudomonas aeruginosa.
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