With the rise of CRISPR technology, we're nearing an age of personalized life science therapies across mass populations.
by David Wollenberg
The human genome, encoded as DNA, encompasses over three billion chemical base pairs. Ten years ago, sequencing a single genome could cost millions of dollars; soon, it will be possible for under $100. This price tag will continue to fall, eventually putting the cost of sequencing in the same range as a flu shot.
Genetic information gives us more insight each day. It helps us understand how genotypes lead to phenotypes and how genetic defects or mutations can lead to cancer, high cholesterol, and other maladies. We are well on our way to predicting how a specific mechanism of action will work on a specific patient—or how likely an individual is to develop various diseases.
This information is valuable, but it becomes truly disruptive in combination with another recent innovation—easy and affordable genome editing through CRISPR technology.
What is CRISPR?
CRISPR stands for “clustered regularly interspaced short palindromic repeats”—not a very informative acronym for most of us! In simple terms, CRISPR is a genome editing technology that can precisely locate and edit specifically chosen sequences of DNA. It’s based on naturally occurring means by which the protein Cas9 can locate and clip DNA sequences.
The journal Science named CRISPR “breakthrough of the year” in 2015. In addition to its remarkable precision, CRISPR has been lauded for its ease of use and relative low cost. Harvard University’s George Church has remarked, “any molecular biology lab that wants to do CRISPR can.”
Numerous start-ups are already focused on developing CRISPR therapies, and clinical trials of CRISPR-edited white blood cells to boost the immune system have been approved in the US and China. In time, the range of conditions that could be treated by CRISPR or similar technologies will grow to include congenital genetic, oncological, viral, bacterial, and more.
Value chain vulnerabilities
Twenty years ago, the dawn of the internet irrevocably disrupted retail, travel, music, and countless other industries. CRISPR and genomics promise the same kind of shakeup for life sciences—not by copycatting old models using new technology, but by disrupting the underlying value chain that advantages today’s leading companies.
It is no longer science fiction to foresee a future in which an individual’s genome, a cancer cell, or a virus could be sequenced, diagnosed, and edited quickly and affordably. A whole new era of individualized therapy development is on its way. Legacy players that don’t evolve may face the same fate as Britannica, Borders Books, and Blockbuster.
Historically, established players in life sciences have been relatively safe from massive disruption, protected by several major barriers to competition:
- Clinical trial costs. It takes an average of 10 years and $2.6B to bring a new drug to market. Though innovations often come from start-ups and universities, only large firms can afford years of late-phase trials on thousands of patients, followed by global marketing campaigns. They maintain their market leadership by licensing or acquiring early-stage therapies from smaller players and bringing them to market.
- Diagnosis and prescription. Through their massive sales teams, life science leaders invest heavily in building relationships with the medical providers who serve as gatekeepers to outpatient therapies and directly deliver inpatient therapies.
- Dispensing of therapies. Established firms have established huge barriers to entry for new firms through a complex system of rebates and relationships with pharmacy benefit managers and contractual relationships with pharmacies.
Affordable genome sequencing and editing have the potential to disrupt all three of these pillars of competitive advantage for large firms. For example:
- Faster, more affordable trials. CRISPR uses low-cost tools and components, while enabling unprecedented specificity. In the future, clinical trials may be able to prove that a therapy works in a matter of days or weeks, without large sample populations.
- New gatekeepers. Today, doctors are the primary actors for diagnosing and prescribing. But what if the rise of sequencing shifts the site of diagnosis from the health care provider to the sequencing provider? If sequencing companies provide data-driven insight into current or potential future conditions, consumers will seek out such companies as sources of medical advice. The network of legacy call points that life science manufacturers have developed with providers will be disintermediated.
- Vertical integration. Sequencing providers could fully integrate sequencing, diagnosis, and genome editing services. As a result, today’s existing stakeholder relationships could lose their value as barriers to entry.
Though this model wouldn’t apply to every condition, it could draw a major share of the market.
To prepare, companies must build agility into currently slow-moving processes, from how they develop new products to how they engage with providers, payers, and patients. The biggest winners will be those who are best able to leverage data—not just for development but also to support diagnosis and treatment.
For the moment, CRISPR remains an emerging technology, but one thing is certain. The life sciences industry is moving faster than ever, and those who stay on the front lines of change will determine the future.