The Therapeutic Areas Evolving Through Cell and Gene Therapy Innovation

Nicole Brooks
4 days ago
10 min read

Contents

Introduction

Ophthalmology

Ikarovec – Geographic Atrophy

Audiology

Rinri Therapeutics - Sensorineural hearing loss

Respiratory: UK Respiratory Gene Therapy Consortium

Cystic Fibrosis

Surfactant Protein Deficiencies

Summary

References

Introduction

The cell and gene therapy landscape is evolving. Being able to directly edit the genome or subcellular processes isn’t an aspirational dream anymore, but a potentially game-changing class of therapeutics for life-threatening and seriously debilitating diseases. A few examples of approved therapies that hit the headlines in the last couple of years [1]:

Casgevy [2]: The first CRISPR-based gene therapy authorised to treat severe sickle cell disease (SCD) and transfusion-dependent B-thalassemia.
Aucatzyl [3]: An approved CAR-T cell therapy for adults with relapsed/refractory B-cell precursor acute lymphoblastic leukaemia (ALL).
Lenmeldy [4]: the first gene therapy for metachromatic leukodystrophy (MLD), a rare degenerative disease.
Elevidys [5]: The first-ever redosable gene therapy for dystrophic epidermolysis bullosa (skin-blistering disorder).

But these examples only scratch the surface of the breadth and depth of science underway in advanced therapies.

This year, in 2026, Zeeks was fortunate enough to attend ELRIG’s Cell and Gene Therapy conference in Cambridge, hearing from the scientists developing cell and gene therapies about innovations in development across a plethora of diseases and therapeutic indications. In the following blog, we want to highlight the companies and discoveries that stood out in selected therapeutic areas.

The Therapeutic Areas Evolving Through Cell and Gene Therapy Innovation

Ophthalmology

Ikarovec – Geographic Atrophy

Cell and gene therapy in the ophthalmic space is very competitive, with several advanced therapies currently in various stages of development across different diseases.

So, what makes Ikarovec interesting?

Firstly, Ikarovec is developing a gene-based therapy for Geographic Atrophy (GA), a leading cause of irreversible blindness worldwide.

Geographic Atrophy is a chronic, progressive condition that typically occurs in the later stages of AMD. Geographic Atrophy is characterised by the progressive loss of retinal pigment epithelium (RPE) cells and results in a loss of central vision through loss of RPE and photoreceptors in the macular region of the eye. This causes difficulty in recognising faces, driving and reading [6].

There are currently two Food and Drug Administration (FDA) approved treatments for GA:

Izervey (avacincaptad pegol) [7], an anti-complement component 3 (C3) therapy administered monthly or every other month.
Syforve (pegcetacoplan) [8], an anti-complement component 5 (C5) inhibitor administered monthly.

Both show anatomic slowing of disease progression, but in the phase 3 clinical trials this did not translate to overall vision preservation.

Therefore, Ikarovec is developing IKAR-001, a novel dual-acting gene therapy to treat GA. The therapy, IKC159V, is engineered to be delivered to the back of the eye, where it will transduce target cells and instruct them to secrete two clinically relevant proteins [9]:

Pigment Epithelium-Derived Factor (PEDF) and,
A soluble form of the complement cofactor sCD46.

PEDF is shown to reduce apoptosis, proinflammatory cytokine expression and lipofusin accumulation, while sCD46 acts as a cofactor for complement factor I to inhibit both the alternative and classical complement pathways [10].

This approach combines complement and inflammation-targeting with neuroprotection. The proteins act synergistically to reduce oxidative stress and inflammation, protect retinal cells from atrophy, and reduce the risk of conversion from GA to the more rapidly progressing wet-AMD.

The novelty of IKAR-001 lies in its proposed single administration. This is exciting because currently, the approved therapies Izervey and Syforve, must be administered monthly or every other month; therefore, the administration burden for patients could be reduced significantly.

Secondly, in the wet age-related macular degeneration indication (wet-AMD), where most therapies are being developed to inhibit vascular endothelial growth factor (VEGF) to reduce leaky blood vessels and improve short-term vision [11], Ikarovec’s lead therapy for wet-AMD combines a potent anti-VEGF with an anti-Connective Tissue Growth Factor (CTGF) protein to minimise tissue remodelling and fibrosis. Ikarovec is hoping to address both angiogenesis and fibrosis that can still occur in Wet-AMD patients treated with anti-VEGF therapies [12].

*Katie Binley presenting Ikarovec Limited at ELRIG Cell and Gene Therapy 2026.*

Audiology

Rinri Therapeutics - Sensorineural hearing loss

Sensorineural hearing loss affects the sensory cells in the inner ear. These specialised otic cells are the hair cells and the auditory neurons.

Age-related hearing loss is a common condition, and with an ageing population, the number of people suffering from this form of hearing loss will only increase.

In healthy hearing, auditory signals from the outside world are converted in the cochlea via the auditory hair cells that sense sound and the auditory neurons which carry the electrical signal towards the brain. These sensory cells are easily damaged and cannot repair or regenerate, leading to gradual sensorineural hearing loss over time [13].

The standard treatment for hearing loss is predominantly medical devices (hearing aids and cochlear implants). There are currently no treatments for damage to auditory neurons [14].

This is what makes Rinri Therapeutics’ portfolio interesting [15]. Rinri has created a proprietary platform for auditory sensory cell development enabling it to develop a portfolio of cell-based treatments to tackle different aspects of hearing loss.

Its most advanced product, Rincell-1, is a pioneering stem cell derived cell therapy which has been developed to regenerate auditory neurons and has been approved for its first in human trial in the UK. It consists of allogeneic otic neural progenitor cells that can mature in vivo into functional auditory neurons, and is an exciting potential evolution for sensorineural hearing loss from the standard of care. This first-in-human trial investigating Rincell-1’s safety will be conducted in NHS patients who are undergoing cochlear implantation.

*Process for development of otic neural progenitors. Image source:* *Rinri Therapeutics website*

Rinri Therapeutics’ Chief Technology Officer, Dr Terri Gaskell, presented at the ELRIG Cell and Gene Therapy Conference on the company’s development history. It was impressive to hear how far Rinri has evolved from its inception and patent in 2019 to the clinical stage. Terri described the long and winding path of cell therapy development, including:

The importance of gaining investment early in pre-clinical development.
Understanding the market access and health economics of an advanced therapy that can be reimbursed by the healthcare system.
Choosing the most appropriate starting materials (which cell lines to use);
Manufacturing and clinical delivery challenges, for example the need to establish upfront that the product format (vials, volume, etc.) is compatible with clinical delivery
Regulatory understanding and engagement to ensure the therapy has been appropriately assessed before starting clinical development, and the studies you are looking to run are in line with regulators’ expectations.
Knowing your indication, meaning conducting consultations with clinical groups and—most importantly—patient groups.
Choosing the right Contract Development and Manufacturing Organisation.

Terri summarised this winding path beautifully: “Start with the end in mind”.

Respiratory: UK Respiratory Gene Therapy Consortium

Cystic Fibrosis

Now we turn our attention to cell and gene therapies for rare respiratory diseases, in particular, cystic fibrosis and the fantastic presentation by Professor Eric Alton of the UK Respiratory Gene Therapy Consortium.

Cystic fibrosis (CF) is a genetic disorder caused by abnormal epithelial ion transport in the apical membrane of secretory cells, leading to thick, dysfunctional mucus in multiple organs, including the lungs, gastrointestinal tract, pancreas, liver, and reproductive system. This results in several clinical complications, such as excessively salty sweat, pancreatic damage, persistent coughs, and respiratory infections [16].

Over recent decades, treatment has improved through antibiotics, pancreatic enzyme replacement, high-fat diets, physiotherapy, and, in severe cases, heart and lung transplantation. More recently, CFTR modulator therapies have significantly improved lung function, exacerbation rates, weight, and quality of life. However, these treatments are not effective for all genetic mutations, and approximately 10–15% of patients worldwide have genotypes that do not respond to current modulators [17]. To confound matters, access to these therapies is also inconsistent due to their high cost.

For over 25 years, the UK Respiratory Gene Therapy Consortium has pursued a single goal: to establish whether gene therapy can become a clinically viable option for patients with CF.

Professor Alton detailed the Consortium’s development of a Wave 1 product (the CF gene delivered via a liposome), which demonstrated a significant benefit in lung function compared with placebo in the world’s largest CF gene therapy trial. Following this, they developed a novel viral vector to deliver the CF gene to the lungs (Wave 2 product), which reached a first-in-human clinical trial stage last year.

Why has the Consortium focused on lentiviral vectors for respiratory diseases?

Recombinant viral vectors such as adenovirus, adeno-associated virus and Sendai virus have been widely used in CF. However, viral vectors induce an immune response in humans. This is because the viral particles in these vehicles are recognised as foreign material [17]. This neutralising antibody response means that, to date, no viral vector has been successfully administered repeatedly without loss of activity.

For several years, the Consortium has collaborated with the Japanese biotechnology company DNAVEC/ID Pharma to develop a new viral vector platform for cystic fibrosis (CF) gene therapy.

Lentiviral vectors offer the potential for long-term gene expression and low immunogenicity, making them particularly attractive for airway gene delivery; however, traditionally used vectors pseudotyped with VSV-G have shown poor efficiency in transducing airway epithelium unless the tissue is deliberately damaged or epithelial tight junctions are opened.

To overcome this limitation, DNAVEC/ID Pharma proposed pseudotyping the lentiviral vector with the F and HN proteins from Sendai virus to improve airway transduction without epithelial preconditioning. Working closely with DNAVEC/ID Pharma, the Consortium evaluated the SIV-F/HN pseudotyped vector in vivo in mice and in several ex vivo lung models [18].

Lentivirus, Sendai Virus and Sendai-Pseudotyped SIV — *Image source:* *UK Respiratory Gene Therapy Consortium website*

These non-clinical studies demonstrated that the vector can efficiently transduce murine airway epithelium through the apical membrane without chemical preconditioning, achieve stable gene expression for the lifetime of the mouse, and permit repeat administration to nasal and lung epithelium.

The vector also successfully transduces human air–liquid interface cultures and lung slices ex vivo, producing stable long-term expression and functional CFTR chloride channels in vitro. Across all tested models, lentivirus-mediated gene transfer achieved levels several orders of magnitude higher than earlier Wave 1 gene therapy products.

Thus, there is hope that as lentiviruses integrate into the host genome, they support prolonged and stable expression after a single dose and do not trigger a strong immune response upon repeat administration.

Surfactant Protein Deficiencies

The Consortium drew on findings from CF studies and sought to apply them to one of the rarest of the rare conditions: surfactant protein deficiencies.

At birth, the lungs are filled with fluid, and to take one’s first breath, you must fill the lungs with air. To do this, you need surfactant to expand the alveolar region of the lungs. In newborns with surfactant protein deficiencies (surfactant protein B, to be more specific), surfactant is not produced. The incidence of this is 1-2 babies per year per country, which means extremely rare.

Newborns with surfactant deficiency need to be placed on ventilators from birth; newborns stay on ventilators until genetic testing comes back from the lab confirming surfactant protein B insufficiency. As the only treatment is lung transplantation, which is rarely attempted due to the lack of donor organs and the unstable state of the disease, the life expectancy of these patients is measured in months and most of the time results in ventilators having to be turned off [19].

But what if scientists could put the missing surfactant protein B into a viral vector?

The UK Respiratory Gene Therapy Consortium discovered from the CF programme that the novel lentivirus was capable of producing high levels of secreted proteins. As a result, the consortium is now pursuing development of gene therapy for the ultra-rare surfactant protein B deficiency, as well as α1-antitrypsin deficiency, interstitial lung diseases and the production of antibodies relevant to influenza and other infections [20].

Summary

This blog explores how cell and gene therapies are rapidly transforming treatment across multiple therapeutic areas, moving from aspiration to clinical reality in diseases with significant unmet need. Drawing on insights from the ELRIG Cell and Gene Therapy conference in Cambridge, it highlights innovation in ophthalmology, audiology and respiratory disease, including Ikarovec’s dual-acting gene therapy targeting both inflammation and neuroprotection in geographic atrophy, Rinri Therapeutics’ stem cell-derived approach to regenerating auditory neurons for sensorineural hearing loss, and the UK Respiratory Gene Therapy Consortium’s work advancing lentiviral vectors for cystic fibrosis and ultra-rare conditions such as surfactant protein deficiencies.

Across these examples, a common theme emerges: the shift towards innovative, potentially single-administration treatments that address underlying disease mechanisms, alongside the complex scientific, regulatory and commercial challenges inherent in bringing advanced therapies to patients.