Pharmaceuticals and Biotechnology

The pharma/biotech industry exists in a constant state of change influenced by scientific advancement, government regulation, evolving business practices, and the changing needs of patients. For example, more drug research and development is being outsourced to contract research organizations (CROs), reducing job opportunities at traditional pharma/biotech companies, but creating openings at CROs. Emerging nanoscience/nanotechnology is creating new, more effective ways to deliver drugs to patients as well as increased demand for scientists and researchers proficient in nanoscience/nanotechnology. The aging of the U.S. population is increasing demand for drugs and therapies that treat heart disease, cancer, chronic lower respiratory disease, and stroke. Another major breakthrough is the discovery of the CRISPR-Cas9 Genome Engineering Tool (more commonly known as CRISPR), which has allowed engineers and scientists to edit genes faster, more efficiently, and less expensively than other existing genome editing methods. CRISPR is a "revolutionary, once-in-a-generation tool that offers the real potential to quickly and efficiently achieve what was once thought impossible. Since 2012, the technology has been adopted rapidly, transforming basic research, drug development, diagnostics and agriculture," according to TIME magazine. The development and use of CRISPR technology will increase demand for genetic science professionals in the next decade due to the variety of applications for this tool—although there will be considerable debate about safety and ethical issues relating to the tool. "Scientists have recently learned that the approach to gene editing can inadvertently wipe out and rearrange large swaths of DNA in ways that may imperil human health," according to Vox.com. These are only a few of the many developments and trends that will shape the future of the industry.

COVID-19 and its Aftereffects

COVID-19 is a novel (new) type of human coronavirus that first emerged in late 2019 in China and that caused hundreds of millions of people throughout the world to get sick and more than 6.8 million people (as of April 2023) to die. COVID-19 spreads mainly from person to person, most commonly through respiratory droplets produced when an infected person coughs or sneezes. In addition to causing serious infections and deaths, the COVID-19 pandemic created business closures and job losses. Some employers delayed and even rescinded job offers, and many companies transitioned internships and other experiential programs to online-only experiences. Most employers conducted job and career fairs and other recruiting events online.

The COVID-19 pandemic had a major effect on drug companies and other employers of pharmaceutical/biotechnology professionals. The majority of sales, office support, legal, technical, and regulatory professionals were forced to work from home. Some manufacturing plants halted production, although many stayed opened because they were deemed “essential businesses.” International supply chains were disrupted when countries—such as China and India (major producers of pharmaceuticals)—were negatively impacted by the pandemic. Some large companies such as Eli Lilly and Bristol Myers Squibb suspended or delayed some of their clinical development programs.

In response to the pandemic, many pharmaceutical/biotech companies and governments pursued pandemic- and infectious diseases-related research in order to develop treatments and vaccines for COVID-19. By March 2021, several vaccines had been rolled out and were being distributed throughout the world. Pfizer, Moderna, and Johnson & Johnson were among these vaccines.

Clinical Research Organizations Are Becoming More Popular

Pharmaceutical companies are increasingly outsourcing drug research and development. This has fueled the rise of clinical research organizations (CROs), which are also known as contract research organizations. The Association of Clinical Research Organizations (ACRO) reports that ACRO member companies “employ more than 130,000 people in over 114 countries—two times more than in 2008—and ACRO members conduct the vast majority of worldwide clinical trials each year, involving nearly two million research participants.” The association estimates that clinical research organization industry revenue represents approximately one-third of total pharma/biotech research and development spending. The top five therapeutic areas for clinical research organizations are oncology, central nervous system, infectious disease, metabolic disorders, and cardiovascular disease. CROs are also beginning to play an increased role in vaccine development.

Growing Use of Generic Drugs

In 1984 the passage of the Drug Price Competition and Patent Term Restoration Act, also called the Hatch-Waxman Act, increased generic drug manufacturers’ access to the marketplace. This legislation caused a boom in generic drug sales. Generic drugs make up 91 percent of industry sales, according to the Food & Drug Administration, and comprise 22 percent of total drug spending in the United States (according to the Association for Accessible Medicines). The United States has the largest generics market in the world. In 2022, U.S. sales of generics reached $86.9 billion, according to IMARC, a market research company. The U.S. generics market is expected the market to reach $110.7 billion by 2028. The biggest generic drug manufacturers are Teva Pharmaceuticals USA (with $8.99 billion in generics sales in 2021), Sandoz ($7.50 billion), Viatris ($5.63 billion), Sun Pharma ($4.64 billion), and Fresenius Kabi ($3.72 billion). Some of the major patented drugs that will expire and become generics in the next several years include Revlimid (2025 to 2026), Stelara (2025 to 2026), Eylea (2025 to 2026), Ibrance (2027), Keytruda (2028), and Eliquis (2027 to 2029).

In the United States, managed health care has contributed to the rise in use of generic drugs. Managed-care programs such as HMOs often ask their doctors to prescribe generic drugs in place of more expensive brand-name products. Recent changes to the Medicare program in the United States will also fuel generic drug sales. Under Medicare Part D, through “multi-tiered pricing,” plans can charge patients more for brand-name drugs than for generics. In addition, plans can ask doctors to fill out prior authorization forms in order for patients to obtain branded drugs.

BioSpace.com reports that the patents will expire for more than 190 drugs from 2022 to 2030, and nearly $236 billion in pharmaceutical sales will be put at risk. The large number of expiring patents on branded pharmaceuticals will increase generic drug companies’ revenues. “Big Pharma” companies that produce brand-name drugs usually attempt to extend their drugs’ exclusivity and prevent generic competition. They do this through litigation and by subtly changing the ingredients of a drug to make it eligible for a new or extended patent. Some “Big Pharma” companies are also entering into licensing agreements with generic drug manufacturers.

Some pharmaceutical companies generate a mixed product line of generics and branded drugs. For example, Novartis has a generics division called Sandoz. Some generic companies also sell branded pharmaceuticals. Generic drug companies have also branched out into generic versions of biologic drugs.

More Outsourcing of Pharma/Biotech Department Tasks to Foreign Countries

Technological breakthroughs and rising labor and material costs have prompted many U.S.-headquartered companies to outsource various tasks to foreign countries, where labor and materials are less expensive. Look for the outsourcing of drug discovery, clinical development, and manufacturing to foreign countries to increase in the future. Companies with sales and marketing departments that are registered and marketing in foreign countries will be good options for American workers who are interested in working abroad. Companies that are registered and marketing their products only in the United States will, of course, retain their U.S.-based sales and marketing departments.

The COVID-19 pandemic or a future pandemic may slow this trend to some degree. During the worldwide pandemic, U.S. hospitals and other medical facilities experienced severe shortages of medical equipment (masks, gloves, ventilators, etc.), as well as drugs. Seventy-two percent of the active ingredient manufacturing facilities for medicines sold in the United States are located in other countries, according to the Food and Drug Administration. China and India—which both experienced serious outbreaks of COVID-19—are the biggest suppliers of active pharmaceutical ingredients. “If we have another global pandemic that leads the world to close borders and leads global supply chains to shatter or to break down, we are distinctly vulnerable because we are now so dependent upon globally integrated supply chains,” said Senator Chris Coons in an NPR interview. Other elected officials are advocating for the increase of U.S.-based biopharmaceutical manufacturing, but experts believe this will be a challenge. “To even get to 50 percent of our drugs being made in the U.S., it will take one to two decades and billions of dollars," said John McShane, a managing partner at the health care product consulting firm Validant, in an NPR interview on the topic.

The Growth of Pharmerging Markets

Although revenue continues to grow in the United States, Great Britain, Germany, and other established markets, much stronger growth is predicted in what are called “pharmerging markets,” developing nations that do not have fully-developed pharmaceutical industries. The global pharmerging market size was valued at $1.48 trillion in 2021, and it is expected to exceed $3.21 trillion by 2030, according to Precedence Research. This represents a compound annual growth rate of 8.98 percent from 2022 to 2030. Many U.S.-based pharmaceutical companies and biotech firms have facilities in these countries. In fact, it’s estimated that Novartis, Sanofi, and AstraZeneca derive 25 percent of their sales from emerging markets. Other companies are planning to build facilities to take advantage of fast-growing consumer markets. Pharmerging markets are typically broken into three tiers:

• Tier I countries (China)
• Tier II countries (Brazil, India, Russia, and South Africa)
• Tier III countries (Poland, Argentina, Turkey, Mexico, Venezuela, Romania, Saudi Arabia, Colombia, Vietnam, Algeria, Thailand, Indonesia, Egypt, Pakistan, Nigeria, and Ukraine)

Countries in the Asia Pacific region (especially China) dominate the pharmerging market, holding a market share of 61 percent in 2021, according to Precedence Research. The continued growth of already large populations (in many Asian Pacific countries, but not in China), rising life spans and declining infant mortality rates, increases in healthcare spending, and improvements in the delivery of health care are fueling increases in pharmaceutical spending.

This offers good opportunities for pharma/bio companies, but it doesn't guarantee easy money. Consumers in these countries typically spend at least half their medicine budgets on lower-cost generic drugs—reducing profit margins. But there’s still a lot of money to be made in pharmerging markets.

An Aging Population is Good News for Pharma/Biotech Companies

By 2030 approximately 72.1 million Americans will be 65 and older, according to the Administration on Aging—more than twice the number in 2000. Senior citizens now account for more than 33 percent of pharmaceutical consumption, and their growing numbers can only bode well for the prospective demand for the industry’s products.

Thus, by focusing its research on the five leading causes of death in the United States (excluding deaths from accidents)—heart disease, cancer, COVID-19, cerebrovascular disease (stroke), and chronic lower respiratory disease—the pharma/biotech industry is set to bring to market drugs for which there is an expanding base of demand.

Information Technology, Bioinformatics, and Other Technologies Are Transforming the Industry

Information technology (IT) is used in virtually all aspects of pharma/biotech research and development. Major IT companies are involved in activities ranging from computational biology research, to biomolecular data management, to biological modeling. An emerging field, bioinformatics, stands out as particularly promising in managing and interpreting the masses of data generated by genomic and proteomic research. Bioinformatics refers to the use of advanced databases and computer analysis tools to perform queries and simulations, cross-reference and compare data, archive test results, and collaborate. Because each pharma/biotech company needs a bioinformatics group, this new field has spawned academic programs and is one of the promising new career paths created by the industry. In addition, IT companies continue to spawn generations of supercomputers, drug discovery optimization software, and physician practice management software.

Another emerging field is artificial intelligence (AI), which is technology that can be programmed to make decisions which normally require human thought and act independently of humans. AI is having a significant impact on the pharmaceutical and biotechnology industry. It is being used to automate many lower-level tasks and analyze vast amounts of data that would be a challenge for humans. All of the 10 Big Pharma companies, as well as hundreds of smaller companies, are either working with AI startup companies or developing their own AI technologies to discover and develop new drugs, develop cures for complex and rare diseases, monitor participant drug adherence and dosage practices during clinical studies, find more reliable patients faster for clinical trials, more effectively assess large volumes of clinical data, and for many other purposes. “AI is already redefining biotech and pharma,” according to Healthcare Weekly. “And 10 years from now, Pharma will simply look at artificial intelligence as a basic, everyday technology.”

Blockchain is another technology that is starting to be used in the industry. It is a shared, distributed ledger database that maintains a continuously-growing list of records that cannot be changed without the agreement of all parties who have access to the database (i.e., no central authority or third-party mediator, such as a bank, is involved in verifying the transaction). Each digital transaction is called a block in the chain of records, hence the blockchain moniker. Each chain is encrypted, in part, with data from the previous block to create the encryption. Both private (permissioned) and public (permissionless) blockchain ledgers can be created. Gartner, a global research and advisory firm, reports that the business value added by blockchain will grow to slightly more than $176 billion by 2025, and then surpass $3.1 trillion by 2030. The pharmaceutical/biotechnology industry is using blockchain to better track legal documents, contracts, and other documents; ensure the accuracy and immutability of information when submitting required regulatory filings; increase the privacy of information and level of transparency during clinical trials; record and manage large volumes of data that are created during research and development and the manufacturing process; and accurately track and trace the products it manufactures and ships to retail stores and health care facilities (doing so will reduce the risk of diversion or counterfeiting), as well as track returns from these customers.

Generative artificial intelligence is one of the newest technologies being used in the biopharmaceuticals industry. It is a form of machine learning algorithms that can be used to create new content, including text, simulations, videos, images, audio, and computer code. One of the best-known example of generative AI is ChatGPT, which was released in late 2022 by the San Francisco-based company OpenAI. Here are a few of the ways that ChatGPT and other forms of generative AI may be used in the biopharmaceutical industry:

• more effectively analyze data from clinical trials, research papers, and other sources to fuel drug discovery and development
• improve the design and efficiency of clinical trials
• improve drug safety monitoring and potentially monitor adverse drug reactions
• improve customer serve chatbot technology and potentially improve patient outcomes
• improve the speed of market research and write basic marketing copy

This all sounds great, but it’s important to understand that generative AI is in its early stages and it is only as good as the information that it receives (or that is available online or through other sources) before it creates new content. “Artificial intelligence and iterations of GPT models will revolutionize multiple aspects of pharma, without a doubt,” says Dr. Andree Bates, in “The Implications of Chat GPT for Pharma” at LinkedIn.com. Dr. Bates, the founder of the biopharma AI strategy firm Eularis, goes on to say that “using GPT models like ChatGPT could be an excellent first step for many within the industry to open their eyes to the potential that lies ahead. But it’s not there yet, and we need to take a questioning approach that ensures we use it ethically, reliably, and safely, including dealing with regulation head-on.”

The Emergence of Personalized Medicine

Current drug therapies are targeted toward the largest patient population possible, but as medical science has advanced, it has become clear that a particular drug may have different levels of effectiveness based on an individual’s physiology. In recent years, the genetic personalization of drug therapy has become possible via pharmacogenomics. Modern Pharmaceutical Industry: A Primer, by Thomas M. Jacobsen and Albert I. Wertheimer, reports that “by collecting epidemiologic, genetic, clinical, and genealogical information, it is possible to uncover genes more effectively that predispose persons to disease, enrich the population that is likely to respond to the drug, and eliminate the nonresponder.”

Today, there are more than 160 FDA-approved personalized medicines (which are also known as individualized or precision medicines), and these medicines account for at least a quarter of new drug approvals that the FDA has approved since 2015. “These medicines are shifting the treatment paradigm for patients, enable increasingly precise assessment of which medical treatments and procedures will be best for each patient,” according to Pharmaceutical Research and Manufacturers of America.

Issues such as cost, privacy, and ethical concerns must be resolved before personalized medicine can be fully integrated into health-care treatment approaches. But it’s clear that pharmacogenetics and pharmacogenomics (the study of how an individual’s genetic composition affects the response to drugs) will play an increasingly important role in the health-care and pharma/biotech industries.

Drug companies will still focus on creating drugs that are targeted toward the largest number of people (and that make the most profits), but the use of pharmacogenomics will help companies save time and money during clinical trials.

The Growth of the Medicinals and Botanicals Industry

An expanding subfield of pharmaceuticals is the medicinals and botanicals industry. These firms oversee the complex process of producing extracts of natural substances and the organic and inorganic chemicals used in a majority of modern medicines. Specific formulas for the preparation of these substances can be found in The United States Pharmacopeia and The National Formulary. If a substance has never been produced on an industrial scale or is entirely new, its manufacturer must produce a document verifying that the substance meets the acceptable legal standards of purity and potency. Finished active ingredients, known as batches, are then shipped to the preparation firm awaiting them, where they are then used to produce drugs. These batches must meet the approval of the Food and Drug Administration (FDA) and the customer company as well as match a master batch in regard to purity and strength. The FDA uses Good Manufacturing Practices and frequent inspection to ensure that optimum standards are observed.

Ethnobotany

Plants aren’t just pretty to look at; they can also help us live healthier lives. Approximately 40 percent of commercially available prescription drugs contain plant-derived compounds. As a result, many major companies have begun to invest time and money in the search for potentially lucrative medications. Discoveries include taxol, a Pacific yew derivative used to treat ovarian and breast cancer; vinblastine, for treating Hodgkin’s disease; scopolamine, for treating motion sickness; and Covifenz, the world’s first plant-based COVID-19 vaccine.

Drug Costs Continue to Rise

Despite efforts by some members of Congress and industry watchdogs, prices for many drugs continue to rise. “There were 1,216 products whose price increases during the twelve-month period from July 2021 to July 2022 exceeded the inflation rate of 8.5 percent for that time period,” according to the U.S. Department of Health & Human Services. “The average price increase for these drugs was 31.6 percent. Some drugs in 2022 increased by more than $20,000, or 500 percent.” The American public and many legislators have responded with outrage over these drastic price increases, but pharmaceutical companies often defend these increases as justified—citing rising research and development costs and other factors. Some drug companies are offering coupons, rebates, and other savings strategies that reduce the cost of medications to some extent. In 2022, Congress passed the Inflation Reduction Act, which was designed to reduce the frequency and size of drug price increases. "The law includes several provisions to lower prescription drug costs for people with Medicare and reduce drug spending by the federal government,” according to the Kaiser Family Foundation. Various components of the act’s drug-related provisions will be phased in each year through 2029.

Congress periodically makes a big show of requiring pharmacy executives to testify regarding these dramatic price increases—with the goal of publicly shaming them into lowering prices. A 2016 review by The Associated Press of the list prices of nearly 30 brand-name medications targeted by Congressional investigators found that these “public shaming” events have little effect. In fact, prices for 22 of these drugs did not change at all, two actually increased in price, and only five declined in price. Look for the battle over drug price increases to continue in the next several years.

“The United States provides drug companies with the strongest patent protections in the world, but legal strategies in the pharmaceutical industry…abuse that liberty,” according to researchers commenting on their study of rising drug costs in the journal JAMA Network Open. “Reasonable drug costs for consumers must be balanced with incentives in the pharmaceutical industry to produce innovative drugs that improve and save lives.”

Expiring Patents Are Creating Challenges for “Big Pharma,” and Opportunities for Generic Drug Makers

A patent is the exclusive legal right given to an individual or a pharma/biotech company by the U.S. government to prevent anyone from making, using, or selling a patented process or invention in the United States. The typical patent protection period is 20 years. After this time, the formulary knowledge contained in the patent application becomes part of the public domain. For a highly profitable drug, a pharmaceutical company may try to obtain an extended patent by making slight changes to the chemical composition of the drug (a process called evergreening). They also use litigation to extend the life of their patents, but this approach isn’t always successful. When a drug patent expires, any company can seek approval to manufacture the drug. Companies that produce patent-expired drugs are generic drug manufacturers.

While the expiration of patents is good news for generic drug companies, its bad news for traditional pharmaceutical companies, which now must compete with generic drug makers for profits. A large number of patents for highly profitable drugs are set to expire in the next few years, which is causing traditional pharmaceutical manufacturers (TPMs) to seek ways to offset these losses since many of these drugs generate large percentages of company profits. For example, Humira generated around 43 percent of AbbVie’s total revenues in 2020, according to FiercePharma.com. Some TPMs are developing partnerships with generic drug makers, while others are starting their own generic drug manufacturing departments.

Some generic drug manufacturers are also turning to legal means to challenge the validity of existing patents held by pharmaceutical companies. The number of lawsuits disputing patents of brand-name pharmaceuticals grew to 417 in 2017—up from 324 in 2016, according to FiercePharma. Generic drug makers see lots of dollar signs in these patent challenges, and this practice is expected to continue during the next decade.

The Emergence of Nanomedicine/Nanotechnology

Current methods of drug delivery, such as orally, intravenously, or transdermally, are not always the most effective routes for a particular therapy. Pharma/biotech companies are currently seeking new, efficient ways to deliver drugs and therapies. One way they’re doing this is by using nanotechnology, which is the scientific manipulation of structures and molecules on a nanometer or atomic scale. Nanomedicine is the application of nanotechnology to improve human health. It “involves the identification of precise targets related to specific clinical conditions and choice of the appropriate nanocarriers to achieve the acquired responses while minimizing side effects,” according to Modern Pharmaceutical Industry: A Primer, by Thomas M. Jacobsen and Albert I. Wertheimer. In nanomedicine, nanoparticles (which are smaller than the head of a pin) are used as a carrier or vehicle for the delivery of drugs into the body. The use of nanoparticles will make the delivery of some drugs easier. It may also make drugs more effective and reduce side effects. The “outlook for the applications of nanomedicine in patient care is very promising,” according to Modern Pharmaceutical Industry. Pharma/biotech companies will spend a lot of research dollars on nanomedicine in the next several years.

Interest Is Growing in Biosimilars

According to the U.S. Food and Drug Administration (FDA), a biosimilar is a “biological product that is highly similar to a U.S.-licensed reference biological product notwithstanding minor differences in clinically inactive components, and for which there are no clinically meaningful differences between the biological product and the reference product in terms of the safety, purity, and potency of the product.” Like generic drugs, biosimilars are drugs that are created by pharma/biotech companies after a patent held by another company has expired.

Biosimilars aren’t the same as generic drugs. The term generic drug applies only to small-molecule drugs that are identical to already-approved small-molecule drugs. The active ingredients in biosimilars, however, feature huge molecules with intricate structures. They are created by using biotechnology, through natural sources, or through synthetic biology. Unlike small-molecule drugs, they are hard to replicate in every detail. Production of biosimilars creates close, but not identical copies.

The U.S. Food and Drug Administration has established licensure standards to ensure that biosimilars are safe and effective. In 2015, it approved Zarxio, the first biosimilar product. This cancer-fighting medication is biosimilar to Amgen Inc.’s Neupogen. Many other companies are developing biosimilar products.

So, why is interest in biosimilars growing? Many pharma/biotech companies are looking to biosimilars as lucrative replacement to “blockbuster drugs” that are becoming harder to develop and launch. The global biosimilar market is expected to increase at a compound annual growth rate of 14.1 percent from 2023 (with $9.5 billion in revenue) to 2032 ($34.4 billion), according to a study by Market.US, a consulting and customized market research company. Factors that are fueling growth in the field include the increasing geriatric population (which requires more medications than people in younger demographic groups); the rising number of chronic diseases such as cancer, rheumatoid arthritis, multiple sclerosis, and cardiovascular diseases; and unhealthy habits (e.g., improper diet, long working hours, insufficient sleep), which are decreasing immunity and creating more risk of disease and other negative medical conditions.

Stem Cell Research

The stem cell controversy derives from the potential power of these undifferentiated embryonic cells to become differentiated into virtually any type of cell found in the human body. Scientists have the ability to maintain and focus the development of such cells to replace existing cells that are either cancerous or have lost their capacity to function normally due to accidents and/or disease. This could potentially provide patients suffering from cancer, diabetes, stroke, brain and spinal cord injuries, and diseases associated with aging with a new source of healthy cells. The consequences of successfully implementing this vision have raised enough questions that the National Institutes of Health (NIH) issued a policy in 2000 that allowed some research under strict federal oversight. In August 2001 the Bush administration restricted the policy somewhat, but allowed continued federal funding. Subsequently, the NIH issued updated guidelines to the industry to implement the new policy. In early 2009,the Obama administration overturned the former administration’s restrictions on using federal dollars for embryonic stem cell research, although stem cell research may not be used in human cloning. In 2019, the Trump administration announced new rules that severely limited government funding of research that uses fetal tissue. In 2021, the Biden administration lifted restrictions on the use of fetal tissue for medical research. It’s likely that this issue will continue be a political “hot potato” in the future.

Cloning

Cloning refers to the laboratory replication of genes, cells, or organisms from a single entity. This means that exact copies of genes can be made. Although the Presidential Commission for National Bioethics Advisory Commission, with industry agreement, acknowledged the moral, ethical, and safety consequences of this activity, there is, nevertheless, one strand of cloning research that is supported by the industry.

Therapeutic cloning, or somatic cell nuclear transfer, is the use of undifferentiated cells that are genetically identical to those of a patient. This means they have no potential of incurring rejection. Such cells can develop into new tissues targeted to replace diseased tissues, offering the possibility of promising new treatments for Alzheimer’s, Parkinson’s, heart disease, and many cancers.

Gene Therapy

Gene therapy is the introduction of specific genes created by recombinant DNA technology into a patient’s body to replace defective ones or to suppress the action of a harmful gene. It may one day help patients with severe and life-threatening diseases that do not respond to traditional treatments. Scientists are currently studying several inherited human diseases that involve defective genes. The first clinical gene therapy trials were initiated in 1990, and the field has expanded greatly in recent years. “Gene therapy is currently available primarily in a research setting,” according to the U.S. National Library of Medicine. “The U.S. Food and Drug Administration has approved more than 30 cellular and gene therapy products for sale in the United States.” Diseases such as cancer, hemophilia, neurodegenerative diseases (e.g., Parkinson’s disease and Huntington’s disease), Leber’s congenital amaurosis, severe combined immune deficiency, and chronic granulomatus disorder are currently being treated with gene or cell therapy, but treatments are being explored for more than 100 diseases. Nearly 300 gene and cell therapies are currently in development.

Gene therapy offers great prospects to help improve human health, but it is controversial. The National Cancer Institute (NCI) says that one such issue is related to the “possibility of genetically altering human eggs or sperm, the reproductive cells that pass genes on to future generations.” This type of gene therapy is referred to as germ-line therapy. Germ-line therapy would forever change the genetic makeup of an individual’s descendants. Although gene therapy of all types is done to improve human health, the NCI cautions that “an error in technology or judgment [during germ-line therapy] could have far-reaching consequences.” Another concern is that gene therapy could be improperly used to enhance human capabilities (such as improving memory and intelligence), and that this type of genetic enhancement would be available only to the rich or well connected. The NCI also points out that “some people associate all genetic manipulation with past abuses of the concept of ‘eugenics,’ or the study of methods of improving genetic qualities through selective breeding.”

Synthetic Biology

Synthetic biology is the use of science and engineering (specifically, gene-sequence information, synthetic DNA, and other technology) to develop biologic entities such as enzymes, cells, and genetic circuits that are not already found in nature. It is also involved with the redesign of existing biologic systems. One example of this was conducted by Jay Keasling, a bioengineer in the Synthetic Biology Department at the University of California, Berkeley. He and his team built and refined a new metabolic pathway in baker’s yeast by assembling genes from three different organisms: the baker’s yeast, gut bacteria, and sweet wormwood. Their goal was to create a synthetic version of the antimalarial drug Artemisinin.

Synthetic biology offers great promise, but some scientists, government officials, and members of the general public are concerned about the potential downside. The main concerns are the accidental release of an organism or system into the environment and the intentional design and release of a harmful organism or system into the environment. Such a synthetic organism might cause unforeseen damage to human, animal, or plant life, or the earth as a whole. Bioethicists also question whether scientists have the right to “tamper with nature” by manipulating genetic code.

In 2012 more than 110 environmental and civil society groups issued the statement, “The Principles for the Oversight of Synthetic Biology,” which called for a global moratorium on the release and commercial use of synthetic organisms until biosafety and government regulations for its use could be established.

Despite these concerns, the synthetic biology industry is growing rapidly.

The global synthetic biology market is expected to reach $39.12 billion by 2027 (up from $13.11 billion in 2022), according to Synthetic Biology Global Market Report 2023 from the Business Research Company. According to the report, the North American market was the largest market for synthetic biology in 2022.