Cell and gene therapy, as a medical affairs discipline
Cell and gene therapy medical affairs, from referral pathway to long-term follow-up
I am a physician and medical affairs manager working on rare disease and advanced therapies in the Gulf. I carried three ex vivo gene therapy programmes at market assessment level and built the diagnostic routes that decide whether a genetic therapy reaches patients. I am not a laboratory scientist. I am the medical affairs lead for the clinical side of a cell and gene therapy launch in these markets.
3 ex vivo gene therapy programmes assessed. 20+ orphan and speciality products. 6 Gulf markets. 100+ key opinion leaders across the Gulf and MENA.
patients in treatment within twelve months, diagnostics first
100+
KOL network, Gulf and MENA
In short
Who you are working with
I am Mahmoud A. Z. Abdelaal, MBBCh, PGD, a physician by training and a medical affairs manager by practice, based in Abu Dhabi. My work is the clinical side of advanced therapies: the patient pathway, the diagnostic gate, the treating centre, the evidence plan, the training of specialist teams, and the follow-up that a one-time therapy implies for the rest of a patient's life.
The gene therapy part of my record is real and should be described accurately. I carried three ex vivo gene therapy programmes at market assessment level: LentiGlobin in sickle cell disease for bluebird bio, and Trimvelis in ADA-SCID and OTL-200 in metachromatic leukodystrophy for Orchard Therapeutics. For Alnylam, Vertex and bluebird bio I introduced technological awareness for those modalities before launch. My launch record sits in the surrounding therapy areas: sickle cell disease, cystic fibrosis and acute hepatic porphyria. I have not carried a gene therapy launch end to end, and I would rather say that than imply otherwise.
I am a physician by training and a generalist in advanced therapies by reading, not a laboratory scientist. I cannot design a vector or run an assay, and nobody should hire me to. What I can do is take the clinical questions a novel modality raises and answer them in a health system that has never delivered one: which patients are eligible, which centres can deliver the therapy, who identifies those patients, what the treating teams need to know, and what evidence the payer will want afterwards.
Therapy areas
Therapy areas and modalities I can speak to
The areas where cell and gene therapy is arriving, and what I can honestly say about each: a programme carried, a pathway built, or a market assessed and read closely.
Sickle cell disease
Haemoglobinopathy work is where I know the Gulf best. I carried LentiGlobin, an ex vivo lentiviral gene therapy for sickle cell disease, at market assessment level for bluebird bio, and I worked the amino acid and small molecule side of the same disease through launch and beyond, including Gulf real-world evidence that became a first-author abstract in Blood (ASH 2020). A gene therapy here faces its hardest question: the eligible population sits inside a much larger diagnosed population, and the patients who need it most are often the least likely to reach a transplant capable centre.
Ex vivo gene therapy programme carried at market assessment level
Beta thalassaemia
Thalassaemia and sickle cell disease travel together in this region: the same haematology clinics, the same transfusion and chelation burden, the same premarital screening programmes, often the same families. A gene therapy for transfusion dependent thalassaemia raises questions I have worked through before: who is transfusion dependent, which centres can manage a myeloablative route, and how far a family will travel for a one-time therapy.
Haematology network across the Gulf and MENA, still active
Duchenne muscular dystrophy
I ran a market assessment for a Duchenne therapy in the Gulf: physician meetings to map the diagnostic journey, identification of where sequencing access was blocking early diagnosis, work with diagnostic labs, and a written account of the gaps that later initiatives used. A Duchenne gene therapy meets exactly that pattern in the region: late diagnosis, a fragmented pathway, and a treatment decision made early because the window is narrow. It was an assessment, not a launch, and it is presented as one.
Haemophilia is where in vivo gene therapy is furthest along, and a fair test of what the medical side of a launch has to get right: a small and well organised patient community, treatment centres that already know their patients, inhibitor history and liver health deciding eligibility, and haematologists who ask about durability in years rather than months. I have not run a haemophilia programme. I have spent years inside the Gulf haematology community that treats these patients.
Haematology KOL relationships across the Gulf, still active
Cystic fibrosis
Mutation-specific therapy, so the diagnostic route decides everything. I worked with diagnostic laboratories to broaden next-generation sequencing access, built patient identification pathways, ran physician education on genetics and mechanism, worked with governments to include eligible patients in treatment programmes, and translated patient information for families. More than three hundred patients were in treatment within twelve months, and the pathway outlasted the project. A gene therapy for cystic fibrosis meets the same gate, one mutation class further on.
300+ patients in treatment within twelve months, government coverage secured
AAV and in vivo therapies
AAV is a delivery vehicle rather than a therapy class, and the practical questions a launch raises are clinical: who is antibody positive and therefore not eligible, what happens to a patient with liver disease, how immunomodulation is managed at the treating centre, and how a single infusion is explained to a family weighing it against lifelong therapy. I introduced technological awareness for novel modalities before launch, which is what medical affairs honestly does with a mechanism the field has not used.
Modality awareness work in market assessment and pre-launch phases
Lentiviral and ex vivo therapies
An ex vivo therapy splits the patient pathway in two: collection or mobilisation of cells, then conditioning and reinfusion, with a manufacturing window in between that the patient lives through. That carries clinical consequences medical affairs has to own: who can be collected, what conditioning means for fertility and for a young patient's body, which hospital can run the whole sequence, and what the family is told while they wait. I carried LentiGlobin, Trimvelis and OTL-200 at market assessment level, where those pathway questions surface first.
3 ex vivo gene therapy programmes carried at market assessment
CAR-T and the treatment centre model
CAR-T sits at the edge of this page: a cell therapy made with a vector, and the model every advanced therapy now borrows. An authorised treatment centre rather than a pharmacy, apheresis and a manufacturing slot, a patient identified earlier than the referral pathway was built for, and follow-up that continues long after the infusion. I carried oncology programmes at market assessment level in the Gulf, including CD19-directed and BTK inhibitor assets, and the centre readiness problems have the same shape.
Oncology programmes carried at market assessment level in the Gulf
Tell me about the therapy and the market
Method
What a gene therapy launch needs, medically
Five workstreams I would expect to own or sequence as the medical affairs lead, in the order a launch date forces.
01
Referral pathway and the centre map
Start by finding out where these patients go. A gene therapy is delivered at a small number of specialist centres, so the referral pathway matters more than the prescriber list: which specialist sees the patient first, what makes them think of a genetic therapy, which hospital can deliver it, and how far the family has to travel. In the Gulf that last question crosses borders, and a pathway that ignores it will sit unused.
02
Patient identification through diagnostics
Every therapy on this page is gated by a test: genotype, mutation class, antibody status, liver function, disease stage. I have built identification pathways before, most fully in cystic fibrosis, where work with diagnostic laboratories to broaden sequencing access and clear patient routes put more than three hundred patients into treatment within twelve months. The same work comes first in a gene therapy launch, and it starts with the labs.
03
Treatment centre readiness
The centre, not the physician, is the unit of a gene therapy launch. Cell collection or apheresis, conditioning, the manufacturing slot, the infusion, short term monitoring and the hospital committee that has to approve a new and expensive therapy all have to be ready before the first patient is enrolled, and most of it is a conversation outside medical affairs. My part is to make sure the clinical requirements reach that conversation early.
04
Long-term follow-up and registry participation
A one-time therapy carries an obligation measured in years. Follow-up schedules, safety reporting and registry participation have to be planned before launch rather than assembled later as a post-marketing commitment. I set up a post-launch registry for an ultra rare therapy at launch so treated patients could be followed over time, and I know what that asks of the medical function: a data question worth answering, a centre willing to enter the data, and someone who reports back.
05
Payer evidence and the value story
A single administration with a long horizon is a hard budget conversation, and it is harder where the payer is the state. The medical function has to supply the population, the eligibility criteria and the natural history of the untreated disease, and the outcomes that will be measured afterwards. The real-world evidence plan belongs in that argument from the first day, because colleagues outside medicine cannot defend a therapy on mechanism alone.
Markets
Where I work, and what each market needs
Gulf: United Arab Emirates, Saudi Arabia, Qatar, Kuwait, Bahrain and Oman
The Gulf is not where gene therapies launch first, which is exactly why the medical groundwork decides the outcome. Patients exist, but the route that finds them through diagnosis is thin outside a handful of centres, treatment has to happen in a specialist hospital that may be in another country, and the payer is usually the state. Add premarital screening programmes that identify carriers, and the picture is a large undiagnosed genetic burden with few delivery points. I have covered all six markets and I live in Abu Dhabi.
Italy
Italy has real centres of gravity for gene therapy in Milan and Rome, a national health service that already funds advanced therapies, and registries built into how these therapies are monitored. Medical Affairs is used untranslated there and the field medical role is the informatore scientifico. I hold work rights in Italy, I am learning Italian at A1 through intensive courses, and I am open to Italy based roles as the language develops.
France
France combines strong academic centres in Paris, Lyon and Marseille with a national assessment route, a separate price negotiation and a large medical affairs community. I hold work rights in France, English is C1 for me with Arabic as a native language, and I read French language material as part of my regulatory and scientific reading. I am open to France based roles and to above country roles that work in English.
Questions
Questions I am actually asked about gene therapy
The gene therapy questions recruiters, agencies and companies tend to ask, answered without inflation.
What does gene therapy medical affairs actually do?
The same discipline as any other launch, with a longer tail. Medical strategy and the evidence story, patient identification through diagnostics, the treatment centre and referral pathway, scientific exchange with the specialists who refer and treat, training, registry and follow-up plans, and the clinical argument the payer will read. What changes is where the uncertainty sits: the mechanism is new to most of the field, eligibility is genetic, delivery is concentrated in a few hospitals, and the outcome is measured over years.
Have you worked on a gene therapy launch?
Not end to end, and I will not pretend otherwise. I carried three ex vivo gene therapy programmes at market assessment level: LentiGlobin in sickle cell disease with bluebird bio, and Trimvelis in ADA-SCID and OTL-200 in metachromatic leukodystrophy with Orchard Therapeutics. I introduced technological awareness for those modalities before launch and built the diagnostic pathways a genetic therapy depends on. What I bring is three launches in the surrounding therapy areas, across six Gulf markets.
You are not a laboratory scientist. Why should that not worry a hiring manager?
Because the laboratory questions are not the ones a launch fails on. The vector is the sponsor's science; the launch is a health system problem: which patients are eligible, who finds them, which centre can deliver the therapy, what the specialists believe, what the payer will ask, and what happens to the patient afterwards. I am a physician by training, I read the mechanism literature closely enough to train specialists on it, and I am clear about the line between what I defend and what belongs to the scientific team.
What is the difference between an AAV therapy and a lentiviral therapy?
The short version, from a medical affairs seat. AAV is delivered in vivo, usually by infusion, and mostly stays outside the genome, so durability and immune response stay open questions and pre-existing antibodies or liver status can decide eligibility. Lentiviral vectors work ex vivo: cells are collected, modified outside the body and returned after conditioning, which integrates the sequence and brings the transplant pathway with it. The clinical consequences differ more than the science does: one brings a transplant centre into the patient journey, the other concentrates the questions on liver health and immune management.
Why does a gene therapy launch depend on diagnostics?
Because genotype decides both eligibility and the size of the population. A therapy that treats one mutation class treats a fraction of the diagnosed patients, and the diagnosed population is itself a fraction of the real one. In the Gulf the bottleneck is sequencing access, so the work is with diagnostic laboratories and the physicians who order the test, not the prescriber list. That is the sequence I lived in cystic fibrosis, where more than three hundred patients reached treatment within twelve months.
What does long-term follow-up require from the medical function?
A plan, a registry and a named owner. Follow-up means a schedule the treating centre can keep, safety reporting that does not depend on the patient staying in one place, and data that answers a question rather than filling a form. I set up a post-launch registry for an ultra rare therapy at launch, and I have worked on Gulf real-world evidence in sickle cell disease used in scientific exchange. For a one-time therapy this is not administration; it is the evidence base of the product being built in public.
Which therapy areas can you actually speak to?
Sickle cell disease, beta thalassaemia, Duchenne muscular dystrophy, cystic fibrosis and haemophilia are the areas I can hold a conversation in, with the modalities themselves: ex vivo lentiviral therapy, AAV delivered therapy and the CAR-T centre model. My portfolio record also covers acute hepatic porphyria, hATTR amyloidosis, primary hyperoxaluria, IgA nephropathy, spinal muscular atrophy and familial chylomicronaemia. For thalassaemia and haemophilia the work behind me is the haematology network and the pathway logic, not a programme of my own.
How do we start?
Send me a message with the therapy area, the market and where you are: a candidate therapy in assessment, a launch date that is already fixed, a diagnostic pathway that does not exist yet, or a centre that has never delivered an advanced therapy. I will tell you honestly whether it is something I do and what it involves. If it is a role, LinkedIn carries the full record. I answer from my own address, and your details stay with me.
Tell me about the therapy and the market
A modality, a therapy area and a market is enough to start. If the launch date is fixed and the pathway is not, say so, because that part cannot be compressed. I answer from my own address, usually within a day, and I will tell you honestly if it is not something I do.
