The biotechnology industry is made up of hundreds of innovative start-ups and larger corporations, that bring together biology and technology to advance medicine and ultimately, save and improve lives. The global biotechnology market was valued at US$859.94 billion in 2022 and is expected to be worth an impressive US$1,683.52 billion by 2030. This growth is being powered by strong government support backed by appealing initiatives, advances in approval processes and reimbursement policies, as well as the standardization of clinical trials. With the demand for personalized medicine and orphan drugs growing, the number of innovative biotech companies emerging is rising and boosting the industry’s market value.

In this blog, we have rounded-up some of the most interesting companies to watch out for this year. This list has been formulated based on our knowledge of the industry and is in no particular order but includes both industry leaders and forward-thinking start-ups who are making themselves known.

1. Alnylam

2. Biogen

Background:

Biogen is a leading biotech company focused on the development of therapies for neurological and neurodegenerative diseases, including multiple sclerosis, Alzheimer's disease, and spinal muscular atrophy. The company was founded in 1978 and has since been responsible for several ground-breaking therapies, including the first treatment for multiple sclerosis, Avonex.

One to watch because:

Several of Biogen’s partnerships are expected to bear fruit this year, with Alzheimer’s drug, Lecanemab, in partnership with Eli Lilly, and anti-depressant, Zuranolone, in partnership with Sage Tx, being two of the according to Fierce Pharma.

Among other trials currently in Phase-3, Biogen is also seeking accelerated approval of Tofersen, an experimental therapy being developed in partnership with Ionis for superoxide dismutase 1 (SOD1) ALS. If successful, it would be the first therapy approved for a genetic cause of ALS.

3. BioNTech

Background:

BioNTech specializes in developing personalized immunotherapies for the treatment of cancer and other diseases. The company, founded in 2008, has made significant contributions to the development of mRNA-based vaccines and therapies and shot to prominence in 2020 with its ground-breaking COVID-19 vaccine, Comirnaty, developed in partnership with Pfizer.

BioNTech's success in mRNA technology has made it a prominent player in the biotech industry, with a market capitalization of over $120 billion. The company has also formed partnerships with other leading pharmaceutical companies, including Roche and Sanofi, to further advance the development of mRNA-based therapeutics for a range of diseases.

One to watch because:

Comirnaty was a huge success story during the global pandemic. The company is looking to follow that up with its seasonal influenza vaccine, BNT161, which is currently in Phase-3. BioNTech expects to initiate another five vaccine trials in infectious diseases in 2023.

BioNTech has been investing the proceeds of Comirnaty into oncology and the company has publicly stated it expects to bring its first cancer vaccine to market by 2030. The company recently announced a partnership with the UK Government to accelerate personalized mRNA therapy trials for cancer and infectious disease vaccines, the first of which are due to .

Currently its most promising cancer vaccine is BNT111, an intravenous therapy that encodes bits of four specific cancer antigens in the drug's RNA to fight skin cancer. The therapy is in a phase 2 trial to treat patients with stage 3 and stage 4 melanoma that's estimated to conclude in October of 2024.

4. Insilico

Background:

Insilico Medicine is a pioneering clinical-stage biotech company that leverages artificial intelligence and machine learning to accelerate drug discovery and development. The company was founded in 2014 and has since been at the forefront of AI-driven drug discovery, with a focus on developing therapeutics for cancer, fibrosis, and aging-related diseases.

Insilico has collaborated with several leading pharmaceutical companies and academic institutions, including GlaxoSmithKline and the University of Toronto, to apply its AI-based approach to drug discovery. The company has also secured significant funding, with a recent $255 million Series C financing round bringing its total funding to over $310 million.

One to watch because:

2023 will likely go down as a breakthrough year for Artificial Intelligence (AI) both inside and outside of the life sciences industry. For its leading candidate, a small molecule drug for idiothetic pulmonary fibrosis (IPF) called INS018_055, Insilico Medicine was granted the FDA’s first Orphan Drug Designation for a . The company has wasted no time in initiating a global, randomized multi-site Phase II trial.

Insilico has several other promising preclinical studies and anticipates further IND filings in 2023, including for its MAT2A inhibitor cancer therapy as it aims to realize the potential of AI to both increase efficiency and decrease the cost of drug development.

5. Moderna

Background:

Moderna is a leading biotechnology company that specializes in the development of messenger RNA (mRNA) therapeutics and vaccines. The company, founded in 2010, has made significant contributions to the field of mRNA technology, with a focus on using mRNA to create personalized cancer vaccines and novel therapies for infectious diseases.

Moderna gained worldwide recognition in 2020 for its COVID-19 vaccine, developed in collaboration with the U.S. National Institutes of Health. The vaccine was the second mRNA vaccine to receive emergency use authorization from the FDA and has played a critical role in the global fight against the COVID-19 pandemic.

One to watch because:

Following success of their blockbuster mRNA covid vaccine, Spikevax, Moderna is continuing to build on continuous advances in basic and applied mRNA science, delivery technology, and manufacturing in other spaces. This includes infectious diseases, such as Flu and respiratory syncytial virus (RSV), and immuno-oncology, rare diseases, cardiovascular diseases and auto-immune diseases. Its pipeline is booming with 48 programs, including four in Phase 3: the mRNA-1345, a COVID booster; flu vaccine, mRNA-1010; and its Cytomegalovirus (CMV) vaccine, mRNA-1647.

The company is also testing its personalized cancer vaccine in combination with Merck's Keytruda in patients with melanoma following surgery, which the US Food and Drug Administration recently granted a breakthrough designation.

6. Genmab

Background:

Genmab specializes in the discovery and development of antibody-based therapies for the treatment of cancer. The Copenhagen-based biotech company, founded in 1999, has made significant contributions to the field of cancer immunotherapy, with a focus on developing novel antibody therapies that harness the power of the body's immune system to fight cancer.

Genmab's most notable achievement is the development of Darzalex, a groundbreaking antibody therapy for the treatment of multiple myeloma. The drug has received approval from regulatory agencies around the world and has been a major success for the company, with annual sales of over $3 billion.

One to watch because:

Genmab recently announced an expansion of their global strategic collaboration with BioNTech to develop and commercialize novel immunotherapies for the treatment of cancer patients. Since 2015, the companies have been working on the joint development of bispecific cancer antibodies and together, have several products with clinical trials underway. The latest deal will see Genmab and BioNTech continue to share costs and potential future profits on a 50:50 basis to develop additional novel monospecific antibody candidates for various cancer indications.

The Danish biotech has formed numerous partnerships over the years that have proven successful, the 2021 approval of cervical cancer treatment Tisotumab vedotin with Seagan, being a prime example.

Several of its other successful partnerships involve products that have been outlicenced and are marketed by partners. This includes Multiple myeloma / AL Amyloidsis drug Daratumumab (Janssen), Relapsing Multiple Sclerosis therapy Oftumumab (Novartis), and Thyroid eye disease treatment, Teprotumumab (Roche). Furthermore, Genmab’s technology is used in the development of several partner’s products currently in stage 3 clinical trials, including Global Blood Therapeutics Sickle Cell therapy, Inclacumab, and Novo Nordisk’s Haemophillia A drug, Mim8, as well as several other products in Phase 2.

7. Relay Therapeutics

Background:

Relay Therapeutics is a biotechnology company that focuses on developing novel small molecule therapies for a range of diseases, with a particular focus on cancer. The company, founded in 2016, uses an innovative approach to drug discovery, combining computational and experimental techniques to design and optimize small molecule drugs.

In 2021, Relay Therapeutics made headlines with the announcement of a $680 million collaboration with Eli Lilly and Company to develop small molecule therapies for cancer and other diseases. The deal was one of the largest in the biotech industry that year and demonstrated the company's growing reputation for innovative drug discovery.

One to watch because:

Relay Therapeutics is one of a new breed of drug discovery companies. Its approach, called Motion-Based Drug Designâ„¢, gives it greater insight into a protein to identify a chemical starting point that it can optimize until its development candidate is selected. This efficient approach has allowed it to generate several promising product candidates that address genetically or clinically validated targets that have previously been intractable or inadequately addressed. Its most advanced asset to date is RLY-4008, an oral small molecule inhibitor of fibroblast growth factor receptor 2 ("FGFR2"), which is expected to enter a pivotal study this year in patients with Cholangiocarcinoma ("CCA"), otherwise known as bile duct cancer, expressing FGFR2 mutations. The company is aiming to secure an accelerated approval for RLY-4008 in early 2024.

While its initial focus has been on enhancing small molecule therapeutic discovery in precision oncology, the company believes its approach can be leveraged for a wide variety of therapeutic areas, including genetic diseases.

8. Rocket Pharma

Background:

Rocket Pharma is a US-based biotechnology company that specializes in developing gene therapies for rare genetic diseases. The company, founded in 2015, uses a lentiviral vector-based approach to deliver gene therapies to target cells in the body, with a focus on treating severe and life-threatening conditions. Its multi-platform development approach applies the lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms.

One to watch because:

With its recent acquisition of Renovacor, Rocket further strengthened its leadership in adeno-associated virus (AAV) based cardiac gene therapy and expands its near-term clinical assets for the treatment of heart conditions. AAV vectors are the leading platform for gene delivery for the treatment of a variety of human diseases. AAVs are seen as an ideal vector for gene therapy because they are small viruses that are not known to cause disease in humans on their own. Rocket’s late-stage pipeline includes the LVV-based gene therapy for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer.

Both LV RP-L102 - indicated for FA - and RP-L201 - indicated for LAD-I - have shown strong enough data in late stage, pivotal studies for Rocket to have decided to make a Biologics License Application to the FDA for permission to commercialize each therapy.

Other notable products in its pipeline include RP-A501 for Danaon Disease, RP-L102 for Fanconi Anemia, RP-L201 for Leukocyte Adhesion, RP-L301 for Pyruvate Kinase Deficiency, and RP-L401 for Infantile Malignant Osteopetrosis.

9. Telix Pharmaceuticals

Background:

Telix Pharmaceuticals is an Australian biotechnology company that specializes in developing radiopharmaceuticals for the diagnosis and treatment of cancer. The company, founded in 2015, uses a targeted approach to imaging and therapy, with a focus on developing precision medicine solutions for cancer patients.

One to watch because:

Telix Pharmaceuticals has gained momentum since the approval of TLX591-CDx, the first FDA-approved product of its kind for prostate cancer imaging and has a very promising late-stage pipeline.

The company recently from a Phase III clinical study of its kidney cancer imaging product, TLX250-CDx, which could enable clinicians to better diagnose and manage the disease without the need for invasive procedures. Also in Phase III is its investigational brain cancer imaging agent TLX101-CDx, for which the company expects to file an New Drug Application (NDA) to the FDA during 2023.

10. Vertex Pharmaceuticals

Background:

Vertex Pharmaceuticals specializes in developing innovative therapies for a range of serious diseases, with a particular focus on cystic fibrosis. The biotech company, founded in 1989, has made significant contributions to the field of precision medicine, with a focus on developing targeted therapies that address the underlying genetic causes of disease.

One to watch because:

2023 is shaping up to be a big year for Vertex and their partnership with CRISPR Therapeutics, as the hotly anticipated Sickle-cell drug, Exa-Cel, is expected to become the first ever CRISPR Cas-9 drug approval. The European Medicines Agency (EMA) has validated Vertex's application, indicating that it has met the necessary requirements to proceed with the approval process and Vertex has been preparing for Exa-Cel’s expected commercial launch by building manufacturing capabilities and establishing patient support programs. The therapy's approval would mark a significant milestone in the field of gene editing and offer hope to patients with genetic diseases.

Well established as a market leader in cystic fibrosis, Vertex has two more exciting products in its pipeline in this therapy area. In Phase 3 is its next-in-class triple combination treatment, Vanzacaftor-Tezacaftor-Deutivacaftor, which seeks to address the underlying causes of the disease to deliver even greater benefit to patients. The company is also working on an mRNA-based candidate with Moderna, VX-522, for which the FDA signed off on an in late 2022.

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