Washington, DC – September 25, 2025 – In the wake of striking advances in gene therapy for Huntington’s disease, a biotech company is positioning itself to deliver a complementary (or alternative) treatment: pharmaceutical-grade cannabinoid softgel capsules. MMJ BioPharma Cultivation, a subsidiary of MMJ International Holdings, claims its oral softgel products—MMJ-001 and MMJ-002—could serve as a safer, more accessible approach for many patients unable or reluctant to undergo neurosurgical gene therapies.
The promise and the limits of gene therapy
Recently, researchers at University College London (UCL) reported encouraging results in silencing the mutant Huntington’s gene using a gene therapy approach. The therapy involves infusing viral vectors directly into deep regions of the brain in a procedure lasting 12 to 18 hours, followed by lengthy recovery. While the early data offers hope of slowing disease progression, considerable challenges remain: the invasiveness of brain surgery, risks of inflammation, the cost and infrastructure required, and the limited reach of this method to only a small subset of patients.
These constraints underscore the need for complementary strategies that can be delivered more broadly and less invasively. For the many patients who are not candidates for neurosurgical therapy, or for whom the costs or risks are prohibitive, alternatives with systemic reach and favorable safety profiles would represent a major advance.
MMJ’s oral softgel capsules: what are they and why they matter
MMJ’s proposed solution is an oral cannabinoid softgel — essentially a pill — produced under Good Manufacturing Practice (GMP) standards and regulated under FDA oversight. Its two lead candidates are:
- MMJ-002, targeted at Huntington’s disease
- MMJ-001, for multiple sclerosis (MS)
Both have already received Orphan Drug Designation and Investigational New Drug (IND) status from the FDA, positioning them among the first cannabis-derived oral medicines specifically aimed at neurodegenerative disorders.
MMJ’s rationale is that cannabinoids — suitably formulated and dosed — may reduce neuroinflammation, exert neuroprotective effects, and alleviate motor and cognitive symptoms. Because their delivery is systemic rather than targeted to specific brain sites via surgery, these capsules could in principle reach broader regions of the brain and be scalable to many more patients.
Duane Boise, CEO of MMJ International Holdings, emphasized that gene therapy and cannabinoid softgels “are not either-or.” He acknowledged that gene therapy might revolutionize treatment for some, but argued that the softgel approach offers a realistic path to broad access for the Huntington’s community today.
Regulatory and manufacturing hurdles ahead
MMJ’s softgel program is not without its challenges. A major hurdle is regulatory: the company is still completing a seven-year regulatory process with the U.S. Drug Enforcement Administration (DEA) to secure a bulk manufacturing license for cannabis-derived active pharmaceutical ingredients. Only once that license is approved can the company begin large-scale GMP production of the softgels in support of clinical trials.
Even after manufacturing approval, MMJ must conduct rigorous clinical trials to demonstrate safety, tolerability, efficacy, and dosage parameters. These trials will be critical, not just for regulatory approval, but also for convincing the medical community and payers that cannabinoid therapy can complement or in some cases substitute more invasive approaches.
Why this matters for patients and families
Huntington’s disease is a devastating neurodegenerative disorder affecting roughly 75,000 people across the U.S., U.K., and Europe. Many more carry the gene mutation and face the grim certainty of future progression. Currently available treatments focus mostly on symptom management; no established therapies have been widely proven to slow or halt disease progression.
For many patients and families, the UCL gene therapy news is a beacon of hope. But for those who cannot—or will not—undergo invasive brain surgery, the UCL approach risks remaining out of reach. A patient quoted in the press release, Jack May-Davis, expressed his admiration for the gene therapy breakthrough while acknowledging the hurdles: the therapy’s complexity, cost, and exclusivity.
In that context, oral therapies that can be prescribed and distributed through conventional pharmacies hold enormous appeal. They promise more equitable access, lower infrastructure demands, and a gentler treatment experience—if they can prove safe and effective.
Cautious optimism: what to watch next
The path ahead is challenging, but MMJ’s approach is one to watch. Key milestones will include:
- DEA licensing approval – enabling large-scale manufacturing of cannabis-derived active ingredients for clinical production
- Phase I/II clinical trials – to establish safety and optimal dosing in humans
- Demonstration of efficacy – ideally, slowing disease markers, reducing symptom progression, or improving quality of life
- Long-term safety monitoring – to ensure no unexpected neurological or systemic adverse effects
- Regulatory and reimbursement strategy – to secure FDA approval and eventual adoption into clinical practice
If the softgel capsules succeed, they could open a new therapeutic category for neurodegenerative disease management—where oral cannabinoid therapy complements, or in some cases broadens, the reach of advanced genetic therapies.
Still, it is important to temper hope with realism: experimental gene therapies remain highly precise, targeted, and mechanistic in ways that systemic cannabinoid therapy may not yet match. The real question is not whether one approach will wholly supplant the other, but whether they can coexist in a more complete toolbox against Huntington’s disease.
Conclusion
The recent gene therapy advances at UCL have reinvigorated the Huntington’s research landscape. Yet they also highlight the gap between high-tech breakthroughs and broad patient applicability. MMJ’s effort to deliver oral, GMP-manufactured cannabinoid softgels represents a bold attempt to bridge that gap: a potentially scalable, non-invasive treatment option that could reach far more patients than neurosurgery alone.
If MMJ’s capsules can clear regulatory and clinical hurdles, they could become a powerful complementary therapy in the fight against Huntington’s. For now, patients, clinicians, and the research community will watch closely — hopeful that this new line of treatment can turn the promise of gene therapy into broader, real-world benefit.
