Personalized medicine

Personalized medicine, a growing trend in healthcare that involves tailoring treatments to individual patients based on their unique genetic and molecular profiles, is expected to be more prevalent in the healthcare sector in 2024, according to MicroMD.

Artificial intelligence (AI) has been a helpful tool for gathering bioinformatic data. Bioinformatics is a field within biotech that combines biology, computer science and statistics, and it can aid the progress of the study of personalized, or precision, medicine. News Medical points to the effectiveness of AI in processing more genomic datasets in less time than it would take human researchers.

Analysts at BioPharma Reporter predict that cell and gene therapies, promising forms of precision medicine, will also be top trends in 2024 aided by the increasing availability of genetic data. In December 2023, the US Food and Drug Administration (FDA) approved Casgevy, the first-ever CRISPR-based therapy ever for treating sickle cell disease (SCD). CRISPR is an innovative gene-editing technology with a variety of uses, including targeting and repairing the genetic mutation that causes SCD. Another gene therapy that was granted FDA approval last year was Lyfgenia, a cell-based treatment that can genetically modify a patient’s stem cells to produce healthy, round red blood cells.

In addition to FDA approval, Casgevy has also been given conditional marketing authorization in the United Kingdom, and Vertex Pharmaceuticals (NASDAQ:VRTX), the company that makes the drug, plans to deliver 50 treatment centers in the US and 25 in Europe in 2024. Bluebird Bio (NASDAQ:BLUE), the maker of Lyfgenia, also reported positive momentum following the drug’s launch in its 2024 financial outlook report.

Additionally, Editas Medicine (NASDAQ:EDIT) is currently running trials of its CRISPR-based therapies, Reni-Cel and EdiTHAL. The former is being tested as a treatment option for SCD and transfusion-dependent beta-thalassemia, while the latter is being tested for treating tumors. Intellia Therapeutics (NASDAQ:NTLA) also has several CRISPR-based therapies for treating various genetic diseases in its pipeline.

CRISPR technology can also be used to modify cells used in tissue engineering or bioprinting, creating personalized tissues and organs for therapeutic purposes like transplants. The technology has been extensively researched and developed over the past decade. Recent improvements include a handheld bioprinting device built by a Canadian research team that can print biocompatible structures inside the human body.

Bioprinting holds great potential for treating a range of conditions, including genetic disorders, cancers and diseases previously thought to be incurable. The ability to print organs and tissues will have a major impact on organ transplantation, significantly reducing the wait times for patients needing organs and lowering the risk of organ rejection, since the new organ will be custom-made based on the patient’s genetic makeup to meet their specific requirements.

Cancer immunotherapy

The field of cancer immunotherapy has seen significant advancements in recent years, due in part to advancements in biotechnology, such as cell-based approaches like CAR-T cell therapy to treat certain types of cancer.

CAR-T therapy involves collecting T-cells, a type of white blood cell that helps protect the body against infections and cancers, from a patient’s blood and priming them to fight cancer cells more aggressively than natural T-cells. The cells are genetically engineered to produce a specific chimeric antigen receptor (CAR) that targets a specific protein found in cancer cells. The cells are multiplied in a lab and then injected back into the patient to, essentially, enhance a patient’s immune system. In an interview with CTV Winnipeg, Dr. David Szwajcer, who has been working with the province of Manitoba, Canada, to provide CAR-T therapy since January 2023, said the treatment has cured “a significantly larger number of people” than treatments that were available just five years ago.

While still in its early stages, CAR-T therapy is a promising area of cancer immunotherapy that has attracted significant interest from investors. The BioInformant’s 2024 CAR-T Financing Report showed that, in the last 10 years, companies working on CAR-T therapies have attracted a total of US$6.7 billion in venture capital investments and raised US$5.76 billion through their initial public offerings. EY Global Life Sciences Deals Leader Subin Baral told Pharmaceutical Technology that CAR-T therapies likely hold the most investment potential within personalized medicine, although he clarified that the biggest investment surge is likely a few years away, indicating the potential for long-term market growth.

However, while the FDA has approved six CAR-T therapies for treating certain types of leukemia and lymphoma, studies reveal a roughly 60 percent relapse rate after its administration, and the FDA opened up an investigation in November 2023 looking into the risk of T-cell malignancy and secondary cancers following treatment. Research into the application of CAR-T for other forms of cancers and autoimmune and infectious diseases is ongoing.

For that reason, Subin said he expects more exploration of other therapies like antibody-drug conjugates (ADCs) in the short term, which have an antibody linked to a cytotoxic payload that is released when the ADC binds to its target. Antibody-based therapies, which are designed to have a targeted effect on cancer cells, have also been studied in different stages of clinical development.

Roche (OTCQX:RHHBF,SWX:RO) subsidiary Genentech is currently investigating the effectiveness of its antibody-based drug, tiragolumab, when used in combination with Tecentriq. Genentech’s treatment targets a receptor referred to as TIGIT, shorthand for T-cell immunoreceptor with Ig and ITIM domains, which can sometimes inhibit the body’s immune response, allowing cancer cells to slip past undetected. Tiragolumab reportedly enhances TIGIT’s ability to detect and attack cancer cells, but the efficacy of TIGIT and its potential uses are still being studied, making this trial particularly noteworthy. Gilead Sciences (NASDAQ:GILD) is another company developing an ADC therapy, and it is currently running a Phase III trial of its therapy for lung cancer.

Overall, the development of novel cancer treatments represents an exciting area of research that has the potential to significantly impact patient outcomes and drive growth in the healthcare industry.

Biotech in agriculture

Since world leaders gathered at COP28 to discuss solutions to the climate crisis and sustainable food systems in November 2023, some experts suggested biotech as a potential source of innovative solutions in agriculture. Biotechnology offers the tools to create a more resilient and sustainable food system.

Genetically modified crops that have been engineered to increase yield, enhance nutritional value and exhibit resistance to climate change have the potential to play a significant role in addressing the global food crisis, according to Kaiser Jamil, a biotechnologist and president of the Third World Organization for Women in Science. In her 2012 article for the United Nations, Biotechnology: A solution to hunger?, Jamil points to the potential of biotech in relieving food shortages around the world.

In the 12 years since her article was published, science has come a long way. Many of the same biotechnologies that are improving healthcare translate into agricultural use as well; gene editing technology can be used to develop new crop varieties or modify specific genes in crops to give them more desirable traits such as higher nutritional value or drought tolerance.

Innovations in biotech have led to advancements in precision agriculture, which uses technologies like drones and sensors to optimize farming with the use of fewer pesticides and fertilizers, and vertical farming, which consists of growing crops in vertically integrated, climate-controlled environments. Additionally, cellular agriculture is a form of biotech in which scientists produce lab-grown meat and dairy products. While it’s still early on, it has the potential to lighten the strain of demand for animal products on traditional farms.

Additionally, synthetic biology can be applied to create bio-based alternatives to non-biodegradable materials and to genetically engineer microbes to assist in bioremediation.

Economic factors to consider

Gabe Cavazos of Leerink Partners described October as a brutal month for biotech companies while speaking at this year’s Biotech Showcase, held in San Francisco in mid-January.

However, according to comments made at the conference, the economic landscape appeared much more positive going into 2024 following an influx of mergers and acquisitions in November and December.

“The five M&A deals in December infused US$30 billion back into health care dedicated funds. So, that just provided more fuel for the fire,” Cavazos said during opening remarks.

The final quarter of 2023 saw six of the 10 largest biotech investment deals of the year, with four completed within the final five weeks of 2023. First, AbbVie (NYSE:ABBV) announced its planned acquisition of Immunogen (NASDAQ:IMGN), along with its ADC cancer therapy Elahere for US$10.1 billion, on November 30. AbbVie later announced its plan to buy out Cerevel Therapeutics, a biopharmaceutical company that specializes in therapies for neurological and neuropsychiatric disorders, for US$8.7 billion.

US Federal Reserve Chair Jerome Powell’s comments following the December 13 meeting of the Federal Open Market Committee, which kept interest rates unchanged, increased expectations that there could be interest rate cuts in 2024. This news may have fueled another round of acquisitions, this time by Bristol Myers Squibb (NYSE:BMY), which announced acquisition deals with Karuna Therapeutics (NASDAQ:KRTX) for US$14 billion on December 22 and RayzeBio (NASDAQ:RYZB) just four days later for US$4.1 billion. Both transactions are expected to close within the first half of 2024.

Additionally, the SPDR S&P Biotech ETF (NYSEARCA:XBI) gained 8.26 percent since the meeting as of January 17

“So, all the signs are there that the inflationary pressures are waning — that the interest rate environment’s going to be more conducive, and biotech’s directly correlated to the interest rate environment,” Cavazos said.

So, will that momentum carry into 2024? Analysts are hopeful. EY’s 2024 M&A Firepower Report revealed that the top 25 biopharma companies have US$1.37 trillion to pursue growth opportunities through M&A or other strategic investments.

“Pharma companies have now been putting to work, which is there is a great need to replenish (the) innovation pipeline,” Maha Katabi, a general partner at Sofinnova Investments, said while speaking at the Biotech Showcase. “There’s plenty of innovation in our sector, especially in the hands of biotech companies. And once proof of concept is achieved, that becomes a very rarefied but unique class that pharma is very interested in talking to. And so these conversations are ongoing.”

So far in 2024, five biotech companies have announced IPOs, with the most recent being Kyverna Therapeutics, which is developing cell therapies for autoimmune diseases. Only 19 IPOs were announced throughout all of 2023, so five in less than three weeks could be indicative of a vibrant and innovative sector.

The bottom line

The long-term success of biotech companies will be dependent on their ability to bring valuable healthcare products to market, which in turn relies on their capacity to conduct innovative research and development and secure funding for clinical trials and commercialization. Bringing new therapies to market is a costly and complex process, one that can be difficult for smaller companies to fund without the external capital of Big Pharma.