Biotech firms experiencing notable growth in the genetic diseases sector?

Biotech firms experiencing notable growth in the genetic diseases sector?

In the realm of biotechnology, recent advancements have focused heavily on developing innovative treatments for genetic diseases. These efforts primarily revolve around gene therapy, genome editing, and cutting-edge delivery platforms, targeting rare and complex conditions. Several biotech startups have raised substantial funding in the past two years, supporting clinical development and scaling manufacturing for these therapeutic modalities.

Key recent developments and funding highlights include:

• Klotho Neurosciences: This company is developing gene therapies with a multi-targeted approach for neurodegenerative and age-related genetic diseases. With a strong intellectual property position, Klotho offers 15–20 year exclusivity on its s-KL platform and partners with AAVnerGene Inc. for cost-effective AAV vector production. Klotho is currently in clinical development with gene therapy candidates like OCU410ST for genetic retinal disorders.
• Glycomine: A clinical-stage biotech specializing in a non-gene-editing approach using lipid nanoparticle-mediated delivery of mannose-1-phosphate for PMM2-CDG, a deadly congenital glycosylation disorder. Glycomine recently raised a $115 million Series C round in April 2025 to advance its lead candidate GLM101 into phase 2b trials, showing clinical improvements in patients.
• Editas Medicine: This company commenced a Phase I/II clinical trial in March 2024 for EDIT-301, an in vivo CRISPR-based therapy for sickle cell disorder, demonstrating nearly complete fetal hemoglobin induction.
• CRISPR Therapeutics and Vertex Pharmaceuticals: The joint venture received FDA acceptance for Casgevy (exagamglogene autotemcel), originally for sickle cell disorder but expanding label use into beta-thalassemia.

Technological advancements driving these developments include CRISPR-Cas9 gene editing platforms, viral vectors for precise gene delivery, and lipid nanoparticle (LNP) systems enhancing delivery efficiency. These innovations have attracted increasing investment from private and government entities, fueling a growing biologics market projected to exceed $1 trillion by 2034, with gene and cell therapies playing a central role.

In addition to Klotho Neurosciences and Glycomine, other notable biotech startups making strides in genetic disease treatments include:

• SpliceBio: Based in Barcelona, Spain, SpliceBio secured U.S Food and Drug Administration (FDA) IND clearance in December 2024, the first-ever for a dual-AAV gene therapy in Stargardt disease (inherited macular degeneration). Its lead candidate, SB-007, is for this condition.
• Beacon Therapeutics: With headquarters in the U.K. and U.S.A., Beacon Therapeutics raised $170 million in a series B funding round in July 2024. Its lead candidate, laru-zova (AGTC-501), is for X-linked retinitis pigmentosa (XLRP).
• Alesta Therapeutics: Based in Leiden, Netherlands, Alesta raised €65 million ($70 million) in a series A funding round in January 2025. Its lead candidates include ALE1 for hypophosphatasia (HPP) and ALE2 for a subset of Charcot-Marie-Tooth disease caused by mutations in tRNA synthetases.
• Azafaros: Headquartered in Leiden, Netherlands, Azafaros raised €132 million ($155 million) in a series B funding round in May 2025. Its lead candidate, nizubaglustat, is for Niemann-Pick disease type C (NPC) and GM1/GM2 gangliosidoses.
• Celosia Therapeutics: Based in Sydney, Australia, Celosia raised $10.4 million (A$16.75 million) in a series A funding round in November 2024. Its lead candidate, CTx1000, is for amyotrophic lateral sclerosis (ALS).
• AIRNA Therapeutics: Headquartered in Cambridge, MA, U.S.A., AIRNA raised $155 million in a series B funding round in April 2025. Its lead candidate, AIR-001, targets alpha-1 antitrypsin deficiency (AATD).
• Actio Biosciences: Based in San Diego, U.S.A., Actio raised $66 million in a series B funding round in June 2025. Its lead candidates include ABS-1230 for KCNT1-related epilepsy and ABS-0871 for TRPV4-positive Charcot-Marie-Tooth disease type 2C (CMT2C).

These biotech startups are making significant strides in developing gene therapies, genome editing treatments, and innovative delivery platforms for a variety of genetic diseases. With substantial funding rounds, regulatory approvals, and promising preclinical results, the field is rapidly progressing towards scalable, multi-targeted, and regulatory-approved treatments for complex inherited disorders.

1. Klotho Neurosciences, a biotech startup focusing on gene therapies for neurodegenerative and age-related genetic diseases, is currently in clinical development with OCU410ST, a gene therapy candidate for genetic retinal disorders.
2. Glycomine, a clinical-stage biotech specializing in lipid nanoparticle-mediated delivery of mannose-1-phosphate for PMM2-CDG, a deadly congenital glycosylation disorder, raised a $115 million Series C round in April 2025.
3. Editas Medicine, a company using CRISPR-Cas9 for sickle cell disorder treatment, demonstrated nearly complete fetal hemoglobin induction in a Phase I/II clinical trial started in March 2024.
4. In the field of health-and-wellness, gene and cell therapies, driven by technological innovations like CRISPR-Cas9, viral vectors, and lipid nanoparticle systems, are projected to play a central role in the growing biologics market, expected to exceed $1 trillion by 2034.
5. Medial-conditions like Stargardt disease, X-linked retinitis pigmentosa, hypophosphatasia, Charcot-Marie-Tooth disease, Niemann-Pick disease type C, amyotrophic lateral sclerosis, alpha-1 antitrypsin deficiency, KCNT1-related epilepsy, and TRPV4-positive Charcot-Marie-Tooth disease type 2C are being targeted by various biotech startups for gene therapy and genome editing treatments.

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