When biotech makes Christmas miracles happen — second edition 

Last year, on Christmas Day, we took a step back from the usual biotech coverage to focus on the patients whose lives were changed when an experimental therapy finally worked, biotech miracles. We looked at moments that felt almost impossible: a child born deaf who could suddenly hear, patients who regained vision after years of blindness, and cancers pushed into remission thanks to new approaches. 

For the most part, biotech is defined by long development timelines, incremental progress, and studies that unfold slowly and quietly. Breakthroughs rarely arrive overnight. But every so often, decades of research, the right technology, and the right patient converge into an outcome that feels, for lack of a better word, miraculous. 

As 2025 comes to an end, we wanted to revisit that spirit.  

Biotech miracle #1: Alyssa and the world’s first base-edited CAR-T cells 

When she was 13, Alyssa Tapley from Leicester was diagnosed with a rare and aggressive form of blood cancer, T-cell acute lymphoblastic leukaemia (T-ALL). She underwent intensive chemotherapy followed by a bone marrow transplant, the standard course of treatment for her disease, but the cancer returned. With conventional options exhausted, her medical team at Great Ormond Street Hospital (GOSH) in London looked to an experimental therapy being developed jointly by GOSH and researchers from University College London (UCL). 

That therapy, BE-CAR7, uses donor T cells that have been extensively edited using a precision genome editing technique called base editing. Instead of modifying a patient’s own cells, the UCL/GOSH team started with healthy donor cells and rewrote parts of their DNA so they could safely recognise and attack Alyssa’s leukaemic cells. 

The team used base editing to change individual DNA letters at several key genes: removing the native T-cell receptor to reduce the risk of graft-versus-host reactions, eliminating CD7 to prevent the modified cells from killing each other, and modifying CD52 to help the edited cells survive alongside preparative chemotherapy. A chimeric antigen receptor (CAR7) was also introduced so the cells could seek and destroy CD7-expressing cancer cells. Base editing, which alters single DNA bases rather than cutting the DNA, offered a more precise way to reprogram these donor cells for therapeutic use. 

Alyssa received her first dose of BE-CAR7 in May 2022 as part of a phase 1 clinical study. Within a month, her cancer had become undetectable, allowing her to proceed to a second stem cell transplant to rebuild her immune system. She remains in remission, now returning for routine follow-up rather than intensive therapy. 

This academic-led effort is now expanding: broader data from the trial show that BE-CAR7 induced deep remissions in most children and young adults with relapsed or refractory T-ALL, potentially transforming a biotech miracle into a broader treatment option. 

Biotech miracle #2: Hassan’s new skin 

When he was seven years old, Hassan was admitted to the burn unit of a children’s hospital in Bochum, Germany, covered in painful, raw wounds from head to toe. He wasn’t in a burns unit because of a fire; he had junctional epidermolysis bullosa (JEB), a rare genetic condition that makes the skin so fragile that even minor friction or pressure can cause large blisters and open wounds. By the summer of 2015, the top layer of his skin, the epidermis, had largely disappeared, leaving him vulnerable to infection and, according to doctors at the time, very likely to die. 

Conventional care for JEB is supportive at best: bandaging wounds, managing infections and pain, and trying to prevent new blisters. There’s no cure, and many patients with severe forms of the disease do not survive into adolescence. With conventional options exhausted, Hassan’s parents asked about experimental approaches and connected with a team at the Center for Regenerative Medicine at the University of Modena and Reggio Emilia. In one of the few remaining patches of intact skin, surgeons took a small biopsy and sent it to the Modena lab, where scientists isolated epidermal stem cells and genetically corrected the underlying defect, a mutation in the LAMB3 gene that impairs laminin-332. 

Back in Germany, plastic surgeons grafted laboratory-grown sheets of the corrected skin back onto Hassan’s body over three surgeries in late 2015 and early 2016. Within months, the new epidermis had taken and was regenerating normally. In 2017, the researchers published data showing that the skin was functional and durable, effectively replacing about 80 % of his body’s epidermis. 

In years since, follow-ups have shown that the gene-corrected skin has continued to function without blistering, and that it regenerates as the epidermis naturally renews itself. Five years later, Hassan was reported to be enjoying a normal life: attending school and enjoying regular activities. The genetically engineered skin has shown normal features, including hair follicles and resistance to everyday stresses, and there have been no signs of the graft failing or causing serious side effects. 

The success of this ex vivo gene therapy approach has helped clear the path toward larger clinical trials, such as the Hologene 5 phase 2/3 trial, which is designed to provide a structured framework for producing and transplanting genetically corrected skin for other patients with LAMB3-related JEB. 

Miracle #3: Treating Pompe disease before birth 

Pompe disease is one of those conditions where timing matters because damage starts early. In its most severe form, CRIM-negative infantile-onset Pompe disease, babies produce little to no functional acid α-glucosidase (GAA), the enzyme needed to break down glycogen. Without it, glycogen accumulates in muscle, and the disease can quickly involve the heart and respiratory system. CRIM-negative patients are also more likely to develop a strong immune response against enzyme replacement therapy (ERT), which can blunt its effect. 

The case that made headlines wasn’t a gene therapy or a one-time fix. It involved a family that had already lived through the worst-case scenario twice. According to the New England Journal of Medicine report, the parents had previously lost two children with infantile-onset Pompe disease and cardiomyopathy. During a new pregnancy, genetic testing via chorionic villus sampling confirmed the fetus carried the same pathogenic GAA variants and would be affected. 

What changed in this pregnancy was the decision not to wait until birth to start treatment. Instead, clinicians delivered recombinant human GAA (alglucosidase alfa), the same enzyme used in standard ERT, directly into the fetal circulation through the umbilical vein, under ultrasound guidance.  

The intent was not to solve the genetic cause in utero, but to prevent the classic early injury from developing before postnatal therapy could even begin. After birth, the baby still required what CRIM-negative Pompe patients typically receive: immune tolerance induction to reduce the risk of anti-drug antibodies and continued ERT. In other words, this was an attempt to change the starting conditions, so postnatal care had a better chance of working. 

At the time of publication, the infant’s course looked unusually mild for CRIM-negative infantile Pompe. The report describes normal cardiac findings, normal creatine kinase levels through follow-up, and age-appropriate motor development. It’s a single case, and it doesn’t prove prenatal ERT will work broadly, but it does show feasibility and a plausible mechanism: start enzyme exposure before irreversible damage accumulates, potentially while the fetal immune system is also more tolerant. 

Long timelines, right treatment, right patient 

These biotech miracle stories don’t point to a single breakthrough or a sudden turning point for biotech as a whole. They reflect what progress in this field usually looks like: long development paths, careful experimentation, and advances that only reach a small number of patients at first. 

Still, when a treatment finally works for someone who has run out of options, its impact is immediate and concrete, a useful reminder of what this industry is about.