Linden Lake is focused on molecular medicines: What are they?

Molecular medicines include regenerative cell and gene/nucleotide therapies that leverage the body’s own molecular biology and enable its ability to heal itself by addressing ailments at their source. These are potentially transformational cures for that will dramatically improve the lives of patients in desperate need.

Molecular medicines target causal cellular and molecular defects and phenomena, rather than previous drugs’ observational focus on symptoms and organs. As such, molecular medicine uses physical, chemical, biological, bioinformatic and medical techniques to understand molecular structures and mechanisms, identify fundamental molecular and genetic errors of disease; and to develop new molecular interventions to correct them. Therefore, molecular medicines are often developed to address upstream specific ‘Central-Dogma’-level genetic lesions which play roles in a variety of disease contexts.

A major pillar of molecular medicine is gene therapy. Gene therapy is a treatment that replaces defective, down-regulated or missing genes with normal genes, enabling cells to function in the manner they are meant to. Gene therapy can also be used to introduce a new gene into the body, to cure or improve its ability to fight disease. This technique – the process of which is known as gene transfer – allows doctors to treat a disorder by using genes instead of drugs or invasive surgery.

In contrast, many biologics developed in the 1990s were designed to only treat disease symptoms or attributes and not its underlying genetic cause. Thus, they were developed to treat the often-ill-defined tissue-level traits/phenotypes (i.e., symptoms) of polygenic diseases in a wide range of patients.

Why is now is the time to invest in molecular medicines?

Until now, molecular medicines have been frustratingly stuck in the realm of promising theory and science fiction. A recent confluence of technical breakthroughs, regulatory reforms and FDA approvals is heralding a ‘Golden Age’ of biotechnology.

Accordingly, now is the time to be investing in and building the companies that are developing the next generation of molecular medicines humanity has been so desperately longing for.

Indeed, the opportunity to create new treatments and cures is enormous. An estimated 7,000 rare diseases are caused by a single gene mutation, making them eligible for targeted therapies. Of those, only 5% have FDA approved therapies associated with them. The cost of sequencing a human genome has plummeted from >$1B in 2003 to less than $1,000 today. As a result, we now know the genetic mechanism underlying some 50% of the estimated known rare genetic diseases. New sequencing and data analysis techniques are estimated to likely to uncover the mechanism underlying many of the remaining diseases by 2020.

Why do molecular medicines offer greater potential for healthcare impact?

Traditional drugs/biologics are focused on downstream biological phenotypes and disease effects and are, therefore, developed to be disease/symptom specific. Thus, when they fail in a late stage clinical trials in a certain disease indication, there is often little asset value that can be salvaged or re-purposed in the wake of such a disappointing result.

Molecular medicines, on the other hand, are developed to address upstream specific ‘Central-Dogma’-level genetic lesions which play roles in a variety of disease contexts; they are far more likely to have broader market potential and applicability to more common diseases. Specifically, as general genetic/cell engineering strategies (e.g., gene editing, gene knock in/down, nucleic acid delivery, stem cell technologies) which are optimized in the context of a rare genetic disease involving a very specific yet often universal mechanism of action; such approaches are often equally applicable in a plug-n-play manner for developing genetic/cellular interventions in a more common disease context.