Damage Control, N-of-1 Therapies, and Introducing MitoCast

The Bio Report

Delivering the Goods

Nearly 70 percent of patients with chronic conditions fail to take their medicines as prescribed. This can lead to poor outcomes and greater healthcare costs. Vivani , which has developed implantable drug delivery technology, said it can provide steady drug release for up to six months with a single implant. This not only can address the problem of non-compliance, but can also reduce side effects associated with fluctuating drug release from conventional delivery methods. We spoke to Adam Mendelsohn , CEO of Vivani, about its implantable drug delivery technology, how it works, and why its lead indication is a GLP-1 implant for weight loss and diabetes.

Developing Cancer Therapies for When Damage Control Goes Awry

The DDR pathway plays a critical role in repairing DNA damage in healthy cells that would otherwise cause mutations or cell death. When this pathway is altered and becomes unable to repair this damage, mutated cells can grow out of control and become cancerous. They can also be more resistant to standard chemotherapies and radiation. Aprea Therapeutics is developing therapies to target mutations in genes in this pathway to treat certain cancers.  We spoke to Oren Gilad , president and CEO of Aprea Therapeutics about the DDR pathway, the use of so-called synthetic lethality to treat these cancers, and why Aprea’s approach may result in safer and more targeted therapies.

Swimming in the New Oil

One of the key obstacles to developing new drugs and diagnostics is the lack of access to longitudinal biological and clinical patient data. Culmination Bio , a spin-out of Intermountain Health, is building the largest patient data platform with more than 40 years of de-identified patient electronic health records and biospecimen data. We spoke to Lincoln Nadauld , president and CEO of Culmination Bio, about the data driving the company’s platform, how partners are using its data today, and how it has the potential to accelerate the development of new diagnostics and therapies.

Does This Drug Represent “A Paradigm Shift for Small Molecule Drug Development?”

NAMPT is a critical enzyme that regulates how cells use energy. It can also play an important role in the ability of cancer cells to survive, grow, and spread. While it’s been recognized as a potential target to treat certain cancers, developing NAMPT inhibitors have been challenging because they can disrupt energy metabolism in healthy cells. Remedy Plan Therapeutics believes it’s overcome the challenges of NAMPT inhibition by a unique mechanism of action that can avoid the problem of toxicity. We spoke to Gregory Crimmins , founder and CEO of Remedy Plan Therapeutics, about the role of NAMPT in cancer, the challenges of developing NAMPT inhibitors without toxicity, and why he believes the company has solved the problem.

Thanks for Prevail InfoWorks and The Copyright Clearance Center (CCC) for their support of The Bio Report

RARECast

Making Exomes More Revealing

Standard exome sequencing, which maps the protein coding regions of the genome, will deliver a diagnosis of someone with a rare disease in about 35 percent of cases. Ambry Genetics ' ExomeReveal seeks to improve the diagnostic yield of these tests by adding RNA analysis to exome testing. That can help resolve variants of uncertain significance in about 2 to 3 percent of the cases. Ambry will also perform continuous reanalysis of the results over time to take into account new gene-disease relationships as they are discovered. This provides a diagnosis to about 5 percent of those without an answer. We spoke to Brigette Davis , chief operating officer for Ambry Genetics, about the diagnostic odyssey for people with a rare disease, Ambry’s new ExomeReveal test, and what people can do to accelerate their path to a diagnosis.

How One Patient Organization Catalyzed Drug Development

Monica Coenraads , the mother of a daughter with Rett Syndrome, has played a critical role in catalyzing development of therapies to treat the rare, neurological disease. She co-founded and served as director of research for the Rett Syndrome Research Foundation. She later founded and today serves as CEO of the Rett Syndrome Research Trust . Her work shows how patient organizations can bridge the translational divide and de-risk rare disease drug development for biopharmaceutical companies. We spoke to Coenraads about her experience with Rett syndrome as a mother of a daughter with the condition, how she crafted a scientific agenda for the organizations she founded, and what other rare disease organizations can learn from her experience.

Enabling On-Demand Therapies

Earlier this year, the company gained attention when it reported that developed a customized antisense oligonucleotide to treat a boy with an ultra-rare neurodevelopmental disorder in a year’s time. The company is leveraging AI to develop oligonucleotide medicines on demand. We spoke to Chris Hart , co-founder, president and CEO of Creyon Bio ; about the proof-of-concept achieved with its recent N-of-1 therapy, the business model for Creyon, and the potential for its approach to reduce the time and cost of drug development.

After Driving an N-of-1 Therapy for Her Son, an Advocate Turns to Helping Others

When Yiwei She 's son Leo was diagnosed with a severe neurodevelopmental condition, he was one of only two people known to have the ultra-rare disease. In a year, though, working with Creyon Bio , the family was able to move from the start of research for an ASO to treating Leo with the experimental therapy. We spoke to She, founder of the TNPO2 Foundation , about how her family was able to treat her son Leo with an experimental ASO with relative speed, the work the TNPO2 Foundation is doing to accelerate the diagnosis of other children with ultra-rare conditions, and its efforts to find accessible and affordable pathways to treatments for others.

Thanks to the Global Genes Corporate Alliance for its support of RARECast 

Life Sciences D'n'A

Tackling the New Bottleneck of Drug Discovery

Markus Gershater , chief scientific officer and co-founder of Synthace sits down with Amar Drawid, Ph.D. to discuss Synthace’s AI-based platform that enables drug developers to design complex experiments, run them, and capture and analyze the data all in an automated way.

 Thanks to Agilisium Labs for its support of Life Sciences D'n'A

MitoCast

The Significance of Mitochondria in Medicine and Health

Pioneering researcher Doug Wallace of Penn Medicine, University of Pennsylvania Health System discusses his groundbreaking work in mitochondrial DNA, its implications for human health, and the need for interdisciplinary collaboration to advance a new field of mitochondrial biology and medicine.

Thanks to mitoworld.org for its support of MitoCast