Biotech firms seeing progress in the field of genetic ailments

Biotech firms seeing progress in the field of genetic ailments

Headline: Biotech Companies Make Strides in Developing Treatments for Genetic Diseases

In the realm of biotechnology, 2025 has been marked by significant advancements in the development of treatments for genetic diseases. Here are some notable developments:

SpliceBio, based in Barcelona, Spain, raised $135 million in a series B funding round in June 2025. Their lead candidate, SB-007, is being developed for Stargardt disease, an inherited form of macular degeneration.

Alesta Therapeutics, with headquarters between Leiden and Boston, raised €65 million ($70 million) in a series A funding round in January 2025. The company's pipeline includes two lead candidates: ALE1 for hypophosphatasia and ALE2 for a subset of Charcot-Marie-Tooth disease caused by mutations in tRNA synthetases.

Vico Therapeutics, headquartered in Leiden, Netherlands, raised €54 million ($60 million) in a series B funding round in January 2024. Their lead candidate, VO659, is an allele-preferential ASO for polyglutamine diseases such as Huntington's and spinocerebellar ataxias.

AIRNA Therapeutics, based in Cambridge, MA, U.S.A., raised $155 million in a series B funding round in April 2025. Their flagship program, AIR‐001, targets the PiZ mutation in the SERPINA1 gene responsible for alpha-1 antitrypsin deficiency.

Actio Biosciences, headquartered in San Diego, U.S.A., raised $66 million in a series B funding round in June 2025. Their pipeline focuses on rare neurological conditions caused by well-characterized mutations, with two lead programs: ABS‐1230 for KCNT1-related epilepsy and ABS‐0871 for TRPV4-positive Charcot-Marie-Tooth disease type 2C (CMT2C).

GEMMA Biotherapeutics, based in Philadelphia, PA, U.S.A., raised $34 million in a seed funding round in December 2024. They are developing multiple gene therapy assets targeting rare diseases.

Beacon Therapeutics, a biotech company developing gene therapies for inherited retinal diseases, raised $170 million in a series B round co-led by Forbion a year ago. Their lead program, laru-zova (AGTC-501), is being developed for X-linked retinitis pigmentosa, a severe form of inherited blindness caused by mutations in the RPGR gene. Enrolment for the phase 2/3 trial of laru-zova is now complete.

Celosia Therapeutics, headquartered in Sydney, Australia, raised $10.4 million in a series A funding round in November 2024. Their lead candidate, CTx1000, is a viral gene therapy for amyotrophic lateral sclerosis (ALS) that targets toxic aggregates of TDP-43.

Azafaros, a biotech company headquartered in Leiden, Netherlands, raised €132 million ($155 million) in a series B funding round in May 2025. Their lead asset, nizubaglustat, is a dual-acting azasugar designed to inhibit two key glycosylation enzymes involved in lipid buildup, with two pivotal phase 3 trials set to begin later this year.

Glycomine, headquartered in San Carlos, CA, U.S.A., raised $115 million in a series C funding round in April 2025. Their lead candidate, GLM101, is for phosphomannomutase‐2 congenital disorder of glycosylation (PMM2-CDG).

Character Biosciences, headquartered in Jersey City, NJ, U.S.A., raised $93 million in a Series B funding round in March 2025. Their lead candidates, CTX114 and CTX203, are designed for geographic atrophy in advanced dry age-related macular degeneration (AMD) and intermediate AMD, respectively.

These advancements are part of a broader trend in the biotech industry, with the clinical use of gene and CRISPR therapies expanding for both inherited genetic diseases and other complex conditions. Personalized treatment development and regulatory acceptance are also supporting these novel biotechnologies.

References: 1. The Science Times 2. Genetic Engineering & Biotechnology News 3. The Scientist 4. Nature

1. The biopharma sector is unfolding remarkable progress in drug development for rare diseases, as evident in SpliceBio's initiative focused on Stargardt disease.
2. Gene therapy companies like Alesta Therapeutics, with a pipeline including ALE1 for hypophosphatasia and ALE2 targeting a subset of Charcot-Marie-Tooth disease, demonstrate the potential of these novel treatments.
3. Clinical trials are underway for some of these innovative solutions, such as Beacon Therapeutics' laru-zova, a gene therapy for inherited retinal diseases approaching phase 2/3 trials.
4. Biotech firms, such as Azafaros and Glycomine, are harnessing the power of gene therapies to address complex medical-conditions like lipid buildup and congenital disorders of glycosylation, respectively.
5. With significant funding in biotech companies like Vico Therapeutics, Character Biosciences, GEMMA Biotherapeutics, and Celosia Therapeutics, the future of health-and-wellness appears to be rooted in science-driven advancements in genomics and gene therapy.

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