Beyond Traditional Medicine: Exploring the Revolutionary Therapies Transforming Patient Care
Biotechnology has revolutionized the healthcare industry, and with the increasing demand for advanced therapies, the focus has shifted towards innovative therapeutic modalities. In this blog article, we will explore some of the cutting-edge therapeutic modalities that have the potential to transform patient care.
Gene Therapy
Gene therapy is a therapeutic modality that aims to replace, remove, or repair a patient's defective or missing genes. This therapy involves introducing genetic material into the patient's cells to correct the underlying genetic defect. The approach can be used to treat a range of genetic diseases, including cystic fibrosis, sickle cell anemia, and muscular dystrophy.
The FDA has approved several gene therapies for clinical use, including Zolgensma, Casgevy, and recently Waskyra. Gene therapy has the potential to cure genetic diseases, and as such, it is one of the most promising areas of biotech research.
Beam Therapeutics - is a biotechnology company established in 2017 that specializes in gene editing therapies using proprietary base editing technology. Based in Cambridge, Massachusetts, the company aims to provide long-lasting solutions for patients with severe genetic diseases and certain cancers by precisely editing individual DNA and RNA bases.
The company's base editing technology builds upon the CRISPR gene editing system and utilizes two main types of base editors: cytosine base editors (CBEs) and adenine base editors (ABEs). These editors create a complex with a guide RNA that targets specific DNA sequences. CBEs convert cytosine (C) to uracil (U), which is then read as thymine (T), while ABEs convert adenine (A) to inosine (I), which is interpreted as guanine (G) by cellular machinery. This enables precise and efficient single-base pair modifications.
Beam released proof-of-concept data for their base-editing therapy BEAM-302 against alpha-1 antitrypsin deficiency (AATD) in March 2025. It also announced new positive safety and efficacy data from the Phase 1/2 trial of risto-cel (formerly BEAM-101), an investigational ex vivo base-editing cell therapy for sickle cell disease with severe vaso-occlusive crises. Both therapies were granted orphan drug designation by the FDA, while BEAM-101 also received RMAT FDA designation. This month Beam gained $255M from its equity stake in Orbital Therapeutics after the latter was acquired by BMS.
REGENXBIO - is a biotechnology company founded in 2008, specializing in the development of gene therapies for patients suffering from a variety of genetic diseases and disorders. The company is based in Rockville, Maryland, and utilizes its proprietary NAV Technology Platform to create adeno-associated virus (AAV) vectors for gene delivery.
The NAV Technology Platform comprises over 100 novel AAV vectors, which are used to deliver functional copies of genes to targeted cells. These vectors have shown promise in their ability to efficiently and selectively target specific tissues and cell types while minimizing immune responses. REGENXBIO's AAV vectors are used in gene therapies targeting various diseases, including retinal, metabolic, and neurodegenerative disorders.
REGENXBIO's pipeline includes both internally developed gene therapies and collaborations with other companies and academic institutions. The company's lead product candidate, RGX-314, is being investigated for the treatment of wet age-related macular degeneration (AMD), diabetic retinopathy (DR), and other chronic retinal conditions. This year, REGENXBIO jointly with AbbVie announced their plans to conduct Phase 3 RGX-314 trials.
In late summer the FDA extended the Prescription Drug User Fee Act (PDUFA) date for REGENXBIO’s RGX-121 for MPS II (Hunter Syndrome) to February 8, 2026, after the company submitted longer-term data, with no safety concerns or inspection findings raised. The biotech completed enrollment in its trial (Oct 2025) for RGX-202 gene therapy for Duchenne muscular dystrophy, which has shown a favourable safety profile.
As for collaborations, in January REGENXBIO partnered with Nippon Shinyaku to develop and commercialize two gene therapies for MPS I & II. Additionally, the company secured $250M in a bond agreement with HealthCare Royalty.
Voyager Therapeutics - is a clinical-stage biotechnology company established in 2013, focusing on the development of innovative gene therapies for severe neurological diseases. Based in Cambridge, Massachusetts, Voyager Therapeutics employs its proprietary adeno-associated virus (AAV) capsid discovery platform, TRACER, to design targeted and efficient gene delivery vectors for various therapeutic applications.
The TRACER platform allows Voyager Therapeutics to identify novel AAV capsids capable of effectively penetrating the blood-brain barrier, offering enhanced specificity and distribution within the central nervous system. This targeted approach aims to overcome the limitations of conventional gene therapy vectors and improve therapeutic outcomes for patients with neurological disorders.
Voyager Therapeutics' pipeline includes gene therapies for a range of neurological diseases, such as Parkinson's disease, Huntington's disease, and amyotrophic lateral sclerosis (ALS).
In March, Voyager revealed positive data from the SAD trial of VY7523 for Alzheimer’s disease. Together with VY7523, data for VY1706 (also targeting Alzheimer’s) was presented at AD/PD 2025. On the other hand, Voyager Therapeutics hit a setback in its SOD1 ALS gene therapy program VY9323 after preclinical studies revealed off-target effects from its siRNA payload, delaying the planned mid-2025 IND. Expanding the Alzheimer’s pipeline, Voyager announced the launch of a new program aiming at APOE4. Closing the year, Voyager partnered with Transition Bio to develop small molecules against TDP-43, a common target in neurodegenerative conditions.
Cell Therapy
Cell therapy involves using living cells to treat diseases. This therapeutic modality can be used to replace damaged or diseased cells in a patient's body. Stem cell therapy is a form of cell therapy that involves using stem cells to regenerate damaged tissue or organs.
Cell therapy is being used to treat a range of diseases, including cancer, heart disease, and diabetes. The FDA has approved several cell therapies, including Kymriah and Yescarta, which are used to treat certain types of cancer.
Allogene Therapeutics - is a biotechnology company founded in 2017 that focuses on developing innovative immunotherapies for the treatment of cancer. Based in South San Francisco, California, Allogene Therapeutics is pioneering the development of allogeneic chimeric antigen receptor (CAR) T-cell therapies, which offer a promising alternative to autologous CAR T-cell therapies.
Allogeneic CAR T-cell therapy involves engineering T-cells from healthy donors rather than the patient's own T-cells. These donor-derived T-cells are modified to express specific CARs that target cancer cells and are subsequently expanded to create an "off-the-shelf" treatment option. Allogene's approach aims to overcome the limitations of autologous CAR T-cell therapies, such as lengthy manufacturing times, high costs, and variable product quality.
Allogene Therapeutics is developing off-the-shelf CAR T therapies across oncology and autoimmune disease. Its lead oncology asset, ALLO-501, targets CD19 in relapsed or refractory non-Hodgkin lymphoma, with Phase 1 durable response data reported in February 2025.
In early 2025, the FDA cleared ALLO-329 for Phase 1 trial across autoimmune conditions like lupus nephritis or systemic sclerosis. In April, ALLO-329 was granted FDA Fast Track Designation. Allogene also reported Phase 1 positive data for ALLO-316 in renal cell carcinoma and subsequently reduced staff count by 28% to focus resources on ALLO-329 and ALLO-316.
Notwithstanding the company’s positive momentum in 2025, the monoclonal antibody ALLO-647 for treating large B-cell lymphoma (LBCL) was discontinued following a fatal infection in a clinical study which led to the update of their trial protocol.
Kite Pharma - is a biopharmaceutical company focused on the development of innovative chimeric antigen receptor (CAR) T-cell and T-cell receptor (TCR) therapies for the treatment of cancer. Founded in 2009 and based in Santa Monica, California, Kite Pharma became a subsidiary of Gilead Sciences in 2017.
Kite Pharma's primary expertise lies in engineering T-cells to target and destroy cancer cells. Their proprietary CAR T-cell therapy platform involves isolating and modifying a patient's own T-cells to express specific CARs, which recognize cancer-specific antigens. The engineered T-cells are then expanded and infused back into the patient to combat the cancer cells.
Kite Pharma's flagship product, Yescarta (axicabtagene ciloleucel), was among the first CAR T-cell therapies approved by the FDA and was the first approved for adult patients with relapsed or refractory large B-cell lymphoma after multiple prior treatments. Their pipeline also includes various investigational CAR T-cell and TCR therapies targeting solid tumors and hematological malignancies.
Kite reported positive safety results in Phase 1a trial in May for KITE-363 - a CAR-T cell therapy for LBCL. In October Kite struck a $1.5B deal with a Chinese biotech Pregene to develop its CAR-T portfolio. For the same purpose Kite got set to buy Interius BioTherapeutics for $350M. In September, Kite broke up the agreement with Shoreline Biosciences, valued at $2.3B amidst the Shoreline staff layoff wave.
Juno Therapeutics - is a biopharmaceutical company that specializes in developing novel immunotherapies for the treatment of cancer. Founded in 2013 and based in Seattle, Washington, Juno Therapeutics became a subsidiary of Bristol Myers Squibb (BMS) in 2018.
Juno Therapeutics' primary focus is on developing chimeric antigen receptor (CAR) T-cell and T-cell receptor (TCR) therapies, which utilize the patient's own immune system to target and eliminate cancer cells. Their proprietary platforms involve engineering T-cells to express specific CARs or TCRs that recognize cancer-specific antigens, enhancing the immune system's ability to fight cancer.
Juno’s pipeline includes a range of investigational CAR T-cell and TCR therapies targeting various hematological malignancies and solid tumors. Their lead product candidate, lisocabtagene maraleucel (liso-cel), is a CD19-directed CAR T-cell therapy for the treatment of relapsed or refractory large B-cell lymphoma (LBCL). In 2025, the FDA approved liso-cel under the brand name Breyanzi for marginal zone lymphoma. Phase 3 trial data supported liso-cel as an efficient treatment for LBCL. In May Juno’s German subsidiary was acquired by TQ Therapeutics to support their cell therapy platform.
RNA-based Therapies
RNA-based therapies involve using small RNA molecules to regulate gene expression. These therapies can be used to treat diseases caused by the overexpression of specific genes. RNA-based therapies can be delivered to cells using lipid nanoparticles or viral vectors.
The FDA has approved several RNA-based therapies, including Onpattro and Givlaari, which are used to treat genetic diseases.
Alnylam Pharmaceuticals - is a biopharmaceutical company focused on the discovery, development, and commercialization of RNA interference (RNAi) therapeutics for the treatment of genetically defined diseases. Founded in 2002 and based in Cambridge, Massachusetts, Alnylam Pharmaceuticals is a pioneer in the field of RNAi-based medicine.
RNAi is a naturally occurring cellular process that regulates gene expression by silencing specific mRNA molecules, preventing the production of disease-causing proteins. Alnylam's proprietary RNAi therapeutics are small interfering RNA (siRNA) molecules that harness this process to target and degrade specific mRNA transcripts, offering a novel approach to treating diseases caused by dysfunctional gene expression.
Alnylam Pharmaceuticals has an extensive pipeline of investigational RNAi therapeutics targeting a wide range of diseases, including rare genetic, cardiovascular, and hepatic disorders. Their first approved product, ONPATTRO (patisiran), is an RNAi therapeutic for the treatment of polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adults.
In March, the FDA expanded approval of Alnylam’s Amvuttra (vutrisiran), making it the first and only drug to treat both nerve and heart forms of ATTR-CM — a rare disease where misfolded transthyretin proteins accumulate in the heart and lead to progressive heart failure. The same month the FDA approved Qfitlia, the Alnylam’s and Sanofi’s siRNA therapy for hemophilia A or B, offering once-every-two-months prophylaxis that lowers antithrombin to reduce bleeding by ~90%. In August Alnylam advanced zilebesiran, a cardiovascular remedy, into Phase 3 trials and presented positive Phase 3 results for vutrisiran.
Antibody Therapies
Antibody therapies involve using monoclonal antibodies to target specific cells or molecules in the body. This therapeutic modality can be used to treat a range of diseases, including cancer, autoimmune disorders, and infectious diseases.
The FDA has approved several antibody therapies, including Keytruda, Opdivo, and Herceptin, which are used to treat cancer.
Genmab - is a biotechnology company focused on the development of differentiated antibody therapeutics for the treatment of cancer. Founded in 1999 and headquartered in Copenhagen, Denmark, Genmab has become a global leader in the field of antibody-based therapeutics.
Genmab's expertise lies in their proprietary antibody discovery platforms, DuoBody and HexaBody, which enable the creation of innovative bispecific and hexavalent antibodies. These next-generation antibody technologies allow for the design of therapeutics with enhanced efficacy, specificity, and safety compared to traditional monoclonal antibodies.
The company has a pipeline of antibody therapeutics in various stages of development, targeting a wide range of cancers. Genmab's flagship product, Darzalex (daratumumab), is a CD38-targeting monoclonal antibody approved for the treatment of multiple myeloma in various settings. Additionally, their pipeline includes promising candidates such as epcoritamab, a DuoBody bispecific antibody targeting CD3 and CD20 for the treatment of B-cell malignancies. In late summer, Genmab published positive results of Phase 3 trials for epcoritamab combined with rituximab and lenalidomide in patients with relapsed/refactory lymphoma. Later, this combination got approved by the FDA. Epcoritamab also proved itself efficient in patients with Richter transformation as was concluded in Phase 1b/2 trial in December 2025.
Recently, Genmab has completed an acquisition of a Dutch biotech Merus. Genmab’s rinatabart sesutecan (Rina-S) showed a 50% response rate in advanced endometrial cancer, supporting its FDA Breakthrough Therapy status and ongoing Phase 3 trials. Additionally, Genmab’s Tivdak, an anti-drug conjugate therapy for cervical cancer got approved in Europe and Japan.
Next-Generation Vaccines
Next-generation vaccines are a new class of vaccines that use innovative technologies to stimulate an immune response against specific diseases. These vaccines can be designed to target a range of diseases, including infectious diseases and cancer.
Several next-generation vaccines are currently in development, including mRNA-based vaccines, DNA-based vaccines, and viral vector-based vaccines. mRNA-based vaccines, such as Pfizer-BioNTech and Moderna's COVID-19 vaccines, have shown exceptional efficacy in clinical trials and have been approved for emergency use by the FDA.
CureVac - is a biopharmaceutical company focused on the development of mRNA-based therapeutics and vaccines for the treatment and prevention of various diseases, including infectious diseases and cancer. Founded in 2000 and headquartered in Tübingen, Germany, CureVac has been a pioneer in the field of mRNA technology.
CureVac's proprietary mRNA platform is based on the use of chemically unmodified mRNA molecules to deliver genetic information to cells, instructing them to produce specific proteins that can elicit therapeutic or preventive effects. The company's technology is designed to optimize mRNA stability, translational efficiency, and immunogenicity, offering a versatile and scalable approach to addressing various medical needs.
CureVac's pipeline includes a range of mRNA-based therapeutics and vaccines targeting infectious diseases, such as COVID-19, as well as cancer immunotherapies and protein replacement therapies for rare diseases. Their lead product candidate, CV8102, is an mRNA-based cancer immunotherapy with positive Phase 1 data.
The FDA has cleared the IND application for CVHNLC, CureVac’s investigational therapy for squamous non-small cell lung cancer. In summer CureVac was acquired by BioNTech for $1.25B.
Moderna - is a biotechnology company dedicated to the development of mRNA-based therapeutics and vaccines for a broad range of diseases, including infectious diseases, cancer, and rare genetic disorders. Founded in 2010 and headquartered in Cambridge, Massachusetts, Moderna has emerged as a leading innovator in the field of mRNA technology.
Moderna's proprietary mRNA platform leverages synthetic messenger RNA (mRNA) to instruct cells to produce specific proteins, generating therapeutic or prophylactic effects. Their approach involves optimizing mRNA sequences, delivery systems, and manufacturing processes to create safe and effective medicines that can be rapidly developed and scaled.
Moderna's diverse pipeline spans various therapeutic areas, including infectious diseases, immuno-oncology, and rare diseases. Their flagship product, Spikevax (elasomeran), is an mRNA-based COVID-19 vaccine that has been authorized for emergency use worldwide, demonstrating the power and potential of mRNA technology in addressing global health challenges.
In addition to their COVID-19 vaccine, Moderna is advancing several other mRNA-based candidates, such as mRNA-4157, a personalized cancer vaccine.
In May, Moderna reported strong Phase 3 data for its combined mRNA-1083 COVID-19 and flu vaccine. Building on this momentum, the FDA approved Moderna’s next-generation COVID-19 vaccine, mNexspike (mRNA-1283), for older adults and higher-risk populations. Positive flu vaccine trial results followed in June, and in July the FDA granted full approval to Spikevax for children aged 6 months to 11 years at elevated risk. In oncology, Moderna reported early Phase 1/2 data in October showing its cancer antigen therapy mRNA-4359, combined with Keytruda, achieved a 24% response rate in heavily pretreated melanoma patients. However, the same month brought a major setback when the company’s cytomegalovirus vaccine mRNA-1647 failed a Phase 3 trial, leading Moderna to halt the program and intensifying pressure on its respiratory portfolio.
Inovio Pharmaceuticals - is a biotechnology company focused on the development of innovative DNA-based immunotherapies and vaccines for the treatment and prevention of cancers and infectious diseases. Founded in 1983 and headquartered in Plymouth Meeting, Pennsylvania, Inovio has been at the forefront of DNA-based medicine.
Inovio's proprietary technology platform, ASPIRE (Antigen SPecific Immune REsponses), utilizes synthetic DNA plasmids to deliver genetic information to cells. These plasmids are designed to instruct cells to produce specific proteins, eliciting targeted immune responses that can either combat infections or attack cancer cells. Inovio's DNA-based approach offers advantages such as rapid development, precise immune targeting, and a favorable safety profile.
Inovio's pipeline includes a diverse range of DNA-based immunotherapies and vaccines targeting various cancers and infectious diseases, such as human papillomavirus (HPV)-associated cancers, hepatitis B, and COVID-19.
Inovio started 2025 with renewed ambitions to develop COVID-19 vaccine after a failed attempt in 2022 jointly with AstraZeneca and Wistar Institute. In summer the company announced pricing for a $25M public offering. Most notably, Inovio Pharmaceuticals completed a rolling BLA submission last month to the FDA for INO-3107, a DNA immunotherapy for adult recurrent respiratory papillomatosis, seeking accelerated approval and priority review with a potential decision in mid-2026.
Topic: Next-Gen Tools
