FDA considers first CRISPR gene editing treatment that may cure sickle cell
A potential CRISPR-based cure for sickle cell disease is moving toward FDA approval, following earlier authorization in the UK, and is raising hopes for patients with a condition that is currently managed rather than eliminated. Commenters examine why the one-time treatment is priced in the millions of dollars, pointing to bespoke lab work, recouping R&D on rare diseases, and the way gene therapies are benchmarked against lifetime treatment costs, while also asking how insurers and public health systems might fund it. The conversation broadens to the promise and limits of gene and mRNA therapies, concerns about access and equity, and ethical questions around germline editing and genetic screening.
Existing Approvals and Cost Structure
- The same CRISPR-based sickle cell therapy has already been approved in the UK.
- Estimates put cost around $2M per patient, in line with other gene therapies.
- Some note pricing appears set against lifetime costs of current treatments, not manufacturing cost.
- Example cited: a hemophilia gene therapy priced at $3.5M is said to cost ~$50k to make/administer, with list price tied to replacing $350k/year drugs.
Why Is It So Expensive?
- Process today is bespoke and labor-intensive: cells are taken out, edited via complex protocols, then reinfused.
- Requires high-end labs, strict quality control, and scarce, highly trained staff.
- High prices argued to recoup R&D and failed trials, especially for rare “orphan” diseases with few patients.
- Others stress prices are also about extracting value from being first to market.
- Many expect costs to drop over decades with automation and patent expiry, citing genome sequencing as precedent.
Is It Really a “Cure”? Mechanism and Durability
- Clarification: this therapy doesn’t fix the sickle hemoglobin gene; it boosts fetal hemoglobin (HbF) by disabling a suppressor.
- It requires high-dose chemotherapy upfront and ex vivo editing of blood-forming stem cells.
- Some call it a cure (if symptoms are permanently gone); others insist that unless disease is fully eradicated, it’s “just” a treatment.
- Longevity of effect is considered promising but still uncertain.
Germline Editing and Ethical Concerns
- Strong scientific/public resistance to germline edits; we “don’t understand enough yet.”
- The He Jiankui case is cited as an early, widely condemned example.
- Some argue delay in germline work is itself unethical for those with severe genetic diseases.
- Others propose IVF plus embryo sequencing as a safer alternative for avoiding hereditary disorders.
Malaria, Sickle Trait, and Tradeoffs
- Sickle cell trait confers partial malaria resistance, explaining its persistence.
- Questions raised whether curing sickle cell might increase malaria vulnerability; most responses see this as theoretically real but practically minor given who will access the therapy.
Access, Insurance, and Global Inequity
- Discussion on whether insurers or national systems will pay, with hepatitis C cures given as precedent.
- Single-payer systems can pool risk and negotiate large discounts.
- Even if production were cheap, multi-million-dollar list prices are seen as completely inaccessible to most patients worldwide.
Other Therapies and Adjacent Tech
- mRNA vaccines and AlphaFold are cited as signs of a bright—though possibly expensive—medical future.
- Some argue mRNA platforms are actually cheap and scalable once developed.
- A detailed anecdote describes L-citrulline supplementation greatly improving sickle cell quality of life; others find it plausible but treat it as adjunctive, not curative.
Genetic Screening and Eugenics Debate
- One subthread proposes mandatory DNA screening and restricting or taxing reproduction for carriers of severe genetic diseases.
- Others strongly reject this as eugenics, raising civil-liberties, abuse-of-power, and historical atrocity concerns.