Early pipeline

Acelot is a small private biotech in South San Francisco. It finds drug candidates with a computer-modelling platform it calls RESOLUTE. ACE-2223 is its lead programme and its first drug to reach human testing. The Australian trial is run by a contract research company, Avance Clinical.

Company website ↗

TDP-43 normally helps nerve cells read their genes correctly. In ALS it leaves its usual place in the cell and forms clumps. Acelot says these clumps are found in about 97% of people with sporadic ALS. ACE-2223 is designed to bind the clumped form and pull it apart, so that TDP-43 can work normally again.

Scientific name: Aggregated TDP-43 (small-molecule disaggregator)

The trial (ACE2223-101) is the first time the drug has been given to people. Healthy adults aged 18 to 65 get a single dose, or doses for 7 days, of ACE-2223 or placebo, with doses rising step by step to check safety and how the body handles the drug. Acelot announced the first dose in September 2026. It has not said when people with ALS will be included.

Phase 1a in healthy volunteers, up to 96 people

Acelot is developing ACE-2223 for sporadic ALS, the common non-inherited form, where TDP-43 clumps are almost always present. The current trial only takes healthy adults aged 18 to 65, who drink the drug as a liquid and stay in a clinical unit.

How it's taken: By mouth (liquid in this trial)

Acelot has not given a date for results or for a first trial in people with ALS. The trial registry dates for enrolment have already passed and do not show when results will come.

The only trial is for healthy adults at the Nucleus Network unit in Melbourne. People with ALS cannot join it. We found no expanded access programme.

Trials and programmes

• ACE2223-101 (Phase 1a)ACTRN12626000231347 · recruiting · Melbourne, Australia

Signal measures how often, and how reliably, Acelot tells the public about this drug. It says nothing about whether the drug works. A quiet company isn't necessarily a bad sign, but regular, on-time updates help patients plan.

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

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Amylyx is a Nasdaq-listed company in Cambridge, Massachusetts, which also developed AMX0035 (see its own page). Its main focus is now avexitide, for low blood sugar after weight-loss surgery. AMX0114 is the ALS drug it is currently testing in people, and Amylyx designed it in-house.

Company website ↗

AMX0114 is an antisense oligonucleotide (ASO), a short strand of genetic material that blocks the instructions a cell uses to make one protein, here calpain-2. Calpain-2 is an enzyme that helps take apart axons, the long fibres that connect nerve cells to muscles, and too much of it is thought to drive nerve damage in ALS. In lab and animal studies AMX0114 lowered calpain-2 and improved nerve cell survival, but most drugs that work in the lab or in mice do not work in people.

Scientific name: Calpain-2 (CAPN2) antisense oligonucleotide

LUMINA is the first time AMX0114 has been given to people. It tests four rising doses in groups of 12 people with ALS. The first person was dosed in April 2025; the first three groups (12.5, 25 and 50 mg) were fully enrolled by August 2026, and a fourth, higher dose is planned. The FDA gave the drug Fast Track status in June 2025, which can speed up later review but says nothing about whether it works.

Phase 1 in people with ALS, about 48 people

The trial is open to adults with ALS whether or not they have a known ALS gene, whose first symptoms began less than 24 months ago and whose breathing capacity is at least 65% of normal. Participants can stay on their usual ALS medicines. The drug is given by lumbar puncture, a needle into the lower back.

How it's taken: Spinal injection every 4 weeks

Amylyx has not given a date for results from the higher doses. The trial registry estimates the main data will be collected by February 2027 and the study will finish in October 2027.

The registry lists LUMINA as recruiting at about 14 sites in the US and Canada. Each person has a 1 in 4 chance of getting placebo. The third dose group filled in August 2026, so places depend on the planned fourth group opening. We found no expanded access programme.

Trials and programmes

• LUMINA (Phase 1)NCT06665165 · recruiting · USA (incl. Boston, Philadelphia, Houston, La Jolla), Canada (incl. Montreal, Calgary)

Signal measures how often, and how reliably, Amylyx Pharmaceuticals tells the public about this drug. It says nothing about whether the drug works. A quiet company isn't necessarily a bad sign, but regular, on-time updates help patients plan.

Announced milestones

• Delivered on time Early cohort data from LUMINA (promised for 2025, delivered 5 Dec 2025)
• Delivered on time Present Cohort 1 biomarker data at a medical meeting (promised for First half of 2026, delivered 23 Jun 2026)

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

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Molefy Pharma is a small Madrid company founded in 2024 as a spin-off of CSIC, Spain's national research council, and majority-owned by the ARQUIMEA group. AP-2 is its most advanced drug. In December 2025 the ALS Association gave it about $1 million through a Hoffman award to support early clinical testing.

Company website ↗

In ALS the protein TDP-43 gets extra chemical tags (phosphorylation), leaves the cell nucleus and builds up where it should not be, so nerve cells stop making some proteins they need. AP-2 blocks CK1δ, one of the enzymes that adds these tags, to help TDP-43 return to the nucleus and work normally.

Scientific name: CK1δ (casein kinase 1 delta) inhibitor

Spain's medicines agency authorised the first human trial in March 2026. It gives rising doses to healthy men at Hospital Universitario de La Princesa in Madrid and started in spring 2026. In September 2026 Molefy said results so far looked favourable for safety and that the trial should finish in early 2027. The European Medicines Agency gave AP-2 orphan drug status in October 2025; that is not an approval.

Phase 1a in healthy volunteers, about 72 people

TDP-43 problems are found in almost all people with ALS, so Molefy is aiming at ALS broadly, and later possibly other diseases such as a form of frontotemporal dementia. The current trial only takes healthy volunteers.

How it's taken: By mouth

Molefy says the healthy-volunteer trial should finish in early 2027, followed by a Phase 1b study in a small group of people with ALS. CSIC has said recruitment for that study could start in January 2027; the company itself says "from 2027".

The only trial takes healthy volunteers at one hospital in Madrid. People with ALS cannot join until the planned Phase 1b study opens. We found no expanded access programme.

Trials and programmes

• AP-2 Phase 1a (healthy volunteers)EU CT 2025-524480-21-00 · recruiting · Madrid, Spain

Signal measures how often, and how reliably, Molefy Pharma tells the public about this drug. It says nothing about whether the drug works. A quiet company isn't necessarily a bad sign, but regular, on-time updates help patients plan.

Announced milestones

• Not due yet Finish the healthy-volunteer trial (promised for First half of 2027)

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

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LeonaBio, formerly called Athira Pharma, is a small Nasdaq-listed biotech in Bothell, Washington. Its closely related drug fosgonimeton failed a large Alzheimer's trial in 2024. In 2026 the company shifted its main focus to lasofoxifene, a breast cancer drug, and said it would update the ALS timeline later.

Company website ↗

HGF (hepatocyte growth factor) is a natural signal that helps nerve cells survive and reduces inflammation. Brelgometon is designed to strengthen this signal. The company's earlier drug, fosgonimeton, works the same way and failed to help people with Alzheimer's disease in a Phase 2/3 trial in 2024.

Scientific name: Positive modulator of the HGF/MET neurotrophic system

The only human study gave single doses, or 10 days of daily doses, of brelgometon or placebo to 80 healthy adults in Dallas, and finished in November 2024. In 2025 the company said it would start a trial in people with ALS in late 2025; that did not happen. In May and June 2026 it said a Phase 2 trial would start in the second half of 2026, then in August 2026 withdrew that timing.

Phase 1 done in 80 healthy volunteers; no ALS trial yet

The company is developing brelgometon for ALS in general, not a genetic subgroup. In the healthy volunteer study it was given by mouth as a liquid, once a day.

How it's taken: By mouth

In August 2026 LeonaBio said it would give an update on the ALS trial's timing once enrolment in its breast cancer trial, ELAINE-3, is complete, which it expects in late 2026. It has not given a new date for the ALS trial.

The only trial, in healthy volunteers, has finished. There is no trial open to people with ALS and we found no expanded access programme.

Trials and programmes

• ATH-1105 Phase 1 (healthy volunteers)NCT06432647 · completed · Dallas TX

Signal measures how often, and how reliably, LeonaBio (formerly Athira Pharma) tells the public about this drug. It says nothing about whether the drug works. A quiet company isn't necessarily a bad sign, but regular, on-time updates help patients plan.

Announced milestones

• Dropped Enable dosing of people with ALS (promised for Second half of 2025)
• Dropped Start a Phase 2 trial in ALS (promised for Second half of 2026)

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Cellenkos is a small private company in Houston, founded in 2016, that makes cord blood Treg products for several diseases, including aplastic anaemia, graft-versus-host disease and myelofibrosis. CK0803 is its ALS product. The company says more than 70 people have been treated across all its programmes. The ALS trial is led at Columbia University in New York.

Company website ↗

Regulatory T cells are immune cells that keep inflammation in check, and in ALS they tend to be fewer and weaker. CK0803 uses Tregs from healthy donor cord blood, grown so they carry high levels of homing proteins (CXCR3 and CD11a) that the company says steer them to inflamed areas of the brain and spinal cord. No donor matching or chemotherapy beforehand is needed. We found no published evidence yet that the cells reach the nervous system in people.

Scientific name: Allogeneic cord blood regulatory T cells (CXCR3-high, CD11a-high)

The registered REGALS trial (listed as Phase 1) has a 6-person open-label safety run-in, dosed by January 2024, followed by a randomised, placebo-controlled part for 60 more people whose main goal includes function and survival (CAFS). That part would measure whether CK0803 works, and we will move it to the main table once it is confirmed to have started. So far Cellenkos has not announced that anyone has been randomised: the registry has said "active, not recruiting" since April 2025, in October 2025 the company said a randomised trial was "planned" for 2026, and in October 2026 the expected completion moved from May 2026 to May 2027. The FDA granted Fast Track status in August 2026, which speeds up contact with the agency but is not a judgement that the treatment works.

Phase 1/1b REGALS; 6-person run-in done, placebo part's progress unreported

REGALS takes adults with ALS whose symptoms began no more than 5 years earlier, with an ALSFRS-R score of 36 to 45 and breathing capacity of at least 50%. People on blood thinners or daily aspirin are excluded. Treatment is 100 million cells by drip into a vein, weekly for 4 weeks then monthly for 5 months, as an outpatient.

How it's taken: IV infusion, weekly then monthly

ClinicalTrials.gov estimates the main part of REGALS will finish in May 2027, a year later than previously listed. Separately, Cellenkos said in October 2025 that a randomised placebo-controlled trial was planned for 2026; it has not announced a start.

REGALS has not been recruiting since April 2025. It ran at Columbia University in New York and at Baylor College of Medicine and the Michael E. DeBakey VA Medical Center in Houston. The 2025 paper treated people under compassionate use, but we found no current expanded access programme.

Trials and programmes

• REGALS (CK0803-101-1)NCT05695521 · active not recruiting · New York (Columbia) and Houston (Baylor, Houston VA), USA

Signal measures how often, and how reliably, Cellenkos tells the public about this drug. It says nothing about whether the drug works. A quiet company isn't necessarily a bad sign, but regular, on-time updates help patients plan.

Announced milestones

• Not due yet Start a randomised, placebo-controlled trial of CK0803 (promised for 2026)

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

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Celosia is a small private Sydney company founded in 2022 by Professor Lars Ittner, whose university lab discovered the approach. CTx1000 is its lead drug. In March 2026 it said it was raising US$80 million to take CTx1000 into later trials and that it plans to file a US trial application with the FDA.

Company website ↗

In ALS the protein TDP-43 leaves the cell nucleus and builds up in toxic clumps. Researchers found that a helper protein, 14-3-3θ, sticks mainly to this faulty TDP-43. CTx1000 uses an emptied-out virus (AAV9) to deliver a small piece of 14-3-3θ joined to a "destroy me" tag, so faulty TDP-43 is sent to the cell's recycling system. In mice it stopped and sometimes partly reversed ALS-like disease, even when given late, but most treatments that work in mice do not work in people.

Scientific name: AAV9-delivered TDP-43 degron (14-3-3θ-based binder)

KOANEWA is the first test of CTx1000 in people. It is open-label, so everyone gets the drug, and each person gets a single dose. The first participant was dosed at Macquarie University Hospital in Sydney, announced on March 23, 2026. Celosia has not said how many people have been treated since.

Phase 1b in people with ALS, up to 15 people (Australia)

The trial takes people whose first ALS symptoms began no more than 2 years ago and who have no or low antibodies against the AAV9 virus shell. People with genetic forms of ALS, including C9orf72, are excluded, except those with changes in the TDP-43 gene (TARDBP) itself. The registry does not say how the single dose is given.

How it's taken: Single dose, one time

Celosia has not given a date for results. The trial registry estimates the main 52-week safety data will be collected by December 2027, with long-term follow-up running to 2030.

The registry lists one recruiting site, Macquarie University Hospital in Sydney. Participants must be on stable ALS medicines for 30 days before screening and test low for antibodies to the AAV9 virus. We found no expanded access programme.

Trials and programmes

• KOANEWA (Phase 1b)NCT07401121 · recruiting · Macquarie University Hospital, Sydney, Australia

Signal measures how often, and how reliably, Celosia Therapeutics tells the public about this drug. It says nothing about whether the drug works. A quiet company isn't necessarily a bad sign, but regular, on-time updates help patients plan.

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

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Insmed is a large New Jersey company whose main products are lung medicines (Arikayce and Brinsupri). Gene therapy is a small, newer part of its pipeline; INS1202 is its second gene therapy to reach people, after one for Duchenne muscular dystrophy.

Company website ↗

INS1202 uses AAV9, an emptied-out virus, to carry a short piece of genetic material (a short hairpin RNA) into nerve and support cells. Once there, it keeps destroying the instructions for SOD1, so less of the protein is made, possibly for years after a single dose. Lowering SOD1 is the same idea behind tofersen, which is approved for SOD1 ALS and needs repeated spinal injections. In SOD1 mice INS1202 improved movement and survival, and it protected nerve cells in lab tests with cells from some sporadic ALS patients, but most treatments that work in mice do not work in people.

Scientific name: AAV9-delivered short hairpin RNA against SOD1

The FDA cleared the trial in 2025 and the first person was dosed in January 2026. The first three people, all with sporadic ALS, got the lowest dose. By late October 2026 the trial had moved to a middle dose, enrolling people with sporadic and SOD1 ALS, with a higher dose possible later if it looks safe.

Phase 1 in people with SOD1 or sporadic ALS, about 23 people

The trial takes adults aged 18 to 79 who either carry a disease-causing SOD1 change or have no known ALS gene, have had ALS for no more than 42 months, have an ALSFRS-R score of at least 24 and breathing capacity of at least 50%. Participants take steroid pills around the dose to prevent immune reactions, so they must be able to swallow pills.

How it's taken: Single spinal injection (one time)

On its October 2026 earnings call Insmed said it expects to share initial data from the ALS programme in 2027.

The registry lists ARMOR as recruiting at seven US centres. Everyone in the trial gets the gene therapy; there is no placebo. In late 2026 the middle-dose group was enrolling both people with sporadic ALS and people with SOD1 ALS. We found no expanded access programme.

Trials and programmes

• ARMOR (Phase 1)NCT07290062 · recruiting · Baltimore MD, Columbia MO, Columbus OH, La Jolla CA, Palo Alto CA, Philadelphia PA, Worcester MA

Signal measures how often, and how reliably, Insmed tells the public about this drug. It says nothing about whether the drug works. A quiet company isn't necessarily a bad sign, but regular, on-time updates help patients plan.

Announced milestones

• Not due yet Share initial data from the ARMOR ALS trial (promised for 2027)

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

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The drug was invented by QurAlis, a Cambridge, Massachusetts biotech, as QRL-204. In June 2024 Lilly paid $45 million upfront for worldwide rights, with up to $577 million more in milestone payments, and now runs the clinical trials. Lilly has said little publicly about the programme beyond the trial registrations.

Company website ↗

In most people with ALS a protein called TDP-43 leaves the cell nucleus, where it normally helps edit genetic instructions. Without it, the instruction for UNC13A, a protein needed at the junctions where nerve cells pass signals, is edited wrongly and less working UNC13A is made. LY4256984 is an antisense oligonucleotide (ASO), a short strand of genetic material designed to correct that editing error. Lab studies showed it restored UNC13A, but it has not yet been shown to help people.

Scientific name: UNC13A splice-switching antisense oligonucleotide

The first trial (Lilly code J6I-MC-OWAA) started in August 2025 and tests four rising doses, each in a group of 8 people (6 on the drug, 2 on placebo), given four times over about 3 months. In May 2026 Lilly started a long-term extension for people who have finished the first trial, so the first participants have already been through dosing. Lilly has not announced how many people have been treated.

Phase 1 in people with sporadic ALS, about 32 people

The trial is for adults aged 18 to 75 with sporadic ALS (no known ALS gene), whose symptoms began less than 24 months ago and whose breathing capacity is at least 50% of normal. The drug is given by lumbar puncture, a needle into the lower back. Faulty UNC13A editing is thought to occur in most ALS, so if it works it could apply widely.

How it's taken: Spinal injection, 4 doses over 3 months

Lilly has not given a date for results. The trial registry estimates the main data will be collected by August 2027 and the study will finish in September 2027.

In the main trial, the registry lists sites recruiting in Leuven, Utrecht, Lübeck, Valencia, Barcelona, Montreal and Toronto, with more in Germany and Canada still to open. Each person has a 1 in 4 chance of getting placebo. The extension study only takes people who were in the first trial. We found no expanded access programme.

Trials and programmes

• J6I-MC-OWAA (Phase 1)NCT07100119 · recruiting · Belgium, Netherlands, Germany, Spain, Canada
• OL01 long-term extension (for people from the first trial)NCT07571174 · recruiting · Canada; European sites not yet open

Signal measures how often, and how reliably, Eli Lilly and Company (licensed from QurAlis) tells the public about this drug. It says nothing about whether the drug works. A quiet company isn't necessarily a bad sign, but regular, on-time updates help patients plan.

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

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NRG Therapeutics is a private biotech in Stevenage, England. In September 2025 it raised £50 million ($67 million), led by the Dementia Discovery Fund, with the Novartis Venture Fund and others, to fund human trials in Parkinson's and an early test of whether the drug works in ALS. It has also had grants from Target ALS and the Michael J. Fox Foundation.

Company website ↗

Mitochondria make energy inside nerve cells. When they are under stress, a channel called the mPTP can open and damage the cell. NRG5051 is designed to reach the brain and keep this channel closed. In August 2026 the company said the drug works through a protein called NLRX1.

Scientific name: Mitochondrial permeability transition pore (mPTP) inhibitor, via NLRX1

The NRG5051-101 trial at CHDR in Leiden, the Netherlands, gives healthy adults single or repeated rising doses of NRG5051 or placebo to check safety. The EU registry also lists people with ALS or Parkinson's, but the company has not reported dosing anyone with ALS. In August 2026 it said ALS dosing was due later in 2026; in October it said ALS dosing, including a Phase 2 trial, is planned for 2027. A separate brain-scan study in healthy volunteers began in London in October 2026.

Phase 1 in healthy volunteers, about 118 people planned

The company describes NRG5051 as a possible disease-modifying treatment for sporadic ALS and for Parkinson's disease. So far it has only reported giving it to healthy volunteers.

How it's taken: By mouth

When dosing began in January 2026, NRG Therapeutics said it expected trial results by the end of 2026. In October 2026 it said the trial was finishing and that dosing in people with ALS, including a Phase 2 trial, is planned for 2027.

The trial is at a single research unit in Leiden that takes healthy volunteers. The company has not opened enrolment to people with ALS. We found no expanded access programme.

Trials and programmes

• NRG5051-101 (Phase 1)EU CT 2025-523872-22-00 · recruiting · Leiden, Netherlands

Signal measures how often, and how reliably, NRG Therapeutics tells the public about this drug. It says nothing about whether the drug works. A quiet company isn't necessarily a bad sign, but regular, on-time updates help patients plan.

Announced milestones

• Not due yet Results from the first human trial (promised for 2026)
• Not due yet First doses in people with ALS (promised for 2026)

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

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ProJenX is a small private biotech in New York, set up to develop prosetin, which came out of research at Columbia University supported by the charity Project ALS. The ALS part of its trial is supported by a $1 million Hoffman award from the ALS Association, and the US Department of Defense ALS research programme is listed as a collaborator.

Company website ↗

In ALS, clumps of the protein TDP-43 put nerve cells under stress. Lab research found that an enzyme called MAP4K drives motor neuron death under this kind of stress. Prosetin is designed to reach the brain and spinal cord and block MAP4K, to slow that loss of nerve cells.

Scientific name: MAP4K inhibitor

PRO-101 tested single and repeated doses in healthy volunteers, then 14 days of rising doses against placebo in 41 people with ALS. Those who finished can keep taking prosetin in an open-label extension. In 2024 the FDA lifted a partial hold that had limited which doses could be used in the US. The FDA gave prosetin orphan drug status for ALS in 2020; that is not an approval. ProJenX says it is designing a Phase 2 trial but has not given a date.

Phase 1 in 48 healthy volunteers and 41 people with ALS

Researchers involved describe prosetin as a possible treatment for sporadic ALS, the common non-inherited form. In the trial it is taken by mouth as a liquid, with the dose based on body weight.

How it's taken: By mouth (liquid)

ProJenX presented long-term data at the NEALS meeting in late October 2026 and says it is using the extension data to design a Phase 2 trial. It has not given a start date.

PRO-101 is fully enrolled and no longer taking new people. Participants already in it can stay on prosetin in the extension. We found no expanded access programme.

Trials and programmes

• PRO-101 (Phase 1)NCT05279755 · active not recruiting · Boston MA, San Antonio TX, Montreal (Canada), Utrecht (Netherlands)

Signal measures how often, and how reliably, ProJenX tells the public about this drug. It says nothing about whether the drug works. A quiet company isn't necessarily a bad sign, but regular, on-time updates help patients plan.

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

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QurAlis is a private biotech in Cambridge, Massachusetts, focused on ALS and related diseases. QRL-101 was licensed from Eli Lilly. QurAlis is also developing it for epilepsy. The company says planning for a Phase 2 trial in ALS is under way, depending on new funding.

Company website ↗

In many people with ALS, motor nerves are overexcitable: they fire too easily, which may speed up their damage. Kv7.2/7.3 channels act as brakes on nerve firing, and loss of TDP-43 in ALS can leave nerve cells short of them. QRL-101 is designed to open these channels. An older drug of this type, ezogabine, was used for epilepsy but was withdrawn because of side effects.

Scientific name: Kv7.2/7.3 potassium channel opener

QurAlis has run several Phase 1 studies, mostly in healthy volunteers, testing single and repeated doses, a nerve-excitability study, and a new formulation finished in August 2026. One study gave a single dose to 12 people with ALS in Utrecht, the Netherlands. A Phase 2 trial in ALS is being planned but has no start date.

Phase 1 done in about 200 people, 12 of them with ALS

QurAlis is targeting people with ALS whose motor nerves are overexcitable, which it says is seen in about half of people with ALS. It has not limited the drug to any genetic form.

How it's taken: By mouth

QurAlis's September 2026 presentation says top-line results from the study of a new formulation are expected in the fourth quarter of 2026. It gives no date for a Phase 2 trial in ALS.

Both recent studies have finished, and none is open to people with ALS. We found no expanded access programme.

Trials and programmes

• QRL-101-04 (single dose in ALS)NCT06714396 · completed · Utrecht, Netherlands
• QRL-101-08 (new formulation, healthy volunteers)NCT07421778 · completed · Groningen, Netherlands

Signal measures how often, and how reliably, QurAlis tells the public about this drug. It says nothing about whether the drug works. A quiet company isn't necessarily a bad sign, but regular, on-time updates help patients plan.

Announced milestones

• Delivered late Top-line results from the single-dose ALS study (QRL-101-04) (promised for First half of 2025, delivered 29 Dec 2025)

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

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Rejukon (瑞吉康) is a small private company based in Shanghai since 2022 that works on diseases caused by protein clumping, such as ALS and Parkinson's disease. RJK002 is its lead drug. Most public information comes from Chinese media and partner announcements rather than English-language company press releases.

Company website ↗

RJK002 uses an emptied-out virus (AAV) to deliver genetic instructions into nerve cells. The company says the resulting product can dissolve protein clumps that have already formed and stop new ones forming, restoring normal cell function. It has not published exactly which protein it acts on. The company reports strong survival gains in ALS animal models, but most treatments that work in animals do not work in people.

Scientific name: AAV gene therapy targeting abnormal protein aggregation (target not disclosed)

China's drug regulator allowed a registered trial in May 2024, and the 9-person Phase 1 trial started at Peking University Third Hospital in September 2024; China's trial registry records the first participant enrolled on October 14, 2024. It stopped enrolling in late 2025. Before that, smaller doctor-led studies at Chinese hospitals had dosed their first patient in September 2023. The FDA granted orphan drug status in October 2023, which is not a decision on whether it works.

Phase 1 in people with ALS in China, 9 people

The Phase 1 trial takes adults with sporadic or familial ALS whose symptoms began no more than 2 years ago, with an ALSFRS-R score of at least 30 and breathing capacity of at least 70%. The drug is given once by spinal injection.

How it's taken: Single spinal injection (one time)

The company has not announced a date for results. The trial registry estimates the main data will be collected by November 30, 2026, with follow-up running to 2030.

Both registered studies are listed as active but no longer recruiting. The company said in 2024 it was preparing an application to run trials in the US, but we found no sign that one has been filed. We found no expanded access programme.

Trials and programmes

• RJK002-0001-R (Phase 1)NCT06493279 · active not recruiting · Beijing, China
• Doctor-led studyNCT06454682 · active not recruiting · Fuzhou, China

Signal measures how often, and how reliably, RJK Biopharma (Shanghai Rejukon Biopharm) tells the public about this drug. It says nothing about whether the drug works. A quiet company isn't necessarily a bad sign, but regular, on-time updates help patients plan.

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

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XellSmart (士泽生物) is a private Chinese company that makes off-the-shelf cell therapies from induced pluripotent stem cells for nervous system diseases, including Parkinson's disease, spinal cord injury and ALS. XS228 is its ALS product.

Company website ↗

Induced pluripotent stem cells (iPSCs) are adult cells reprogrammed in a lab so they can become almost any cell type. XellSmart turns donor iPSCs into motor neuron progenitors, early-stage cells that are meant to mature into motor neurons or support existing ones. Because the cells come from donors rather than each patient, they can be made in large batches. Earlier stem cell therapies for ALS have not been shown to work in large controlled trials.

Scientific name: Allogeneic iPSC-derived motor neuron progenitor cells

China's drug regulator approved registered trials in May 2025, and the first participant in the Beijing trial was dosed by early October 2025. The FDA also cleared a US Phase 1 trial in early 2025, but we found no sign it has started. The company calls the Chinese study Phase I/II; ClinicalTrials.gov lists it as Phase 1. The FDA granted orphan drug status in 2023, which is not a decision on whether it works.

Phase 1 in people with ALS in China, about 12 people

The trial takes adults aged 18 to 75 with definite or probable ALS and breathing capacity of at least 70% of normal. People with only upper motor neuron symptoms (primary lateral sclerosis) are excluded. Cells are given by lumbar puncture, a needle into the lower back.

How it's taken: Spinal injection, once or 4 times

XellSmart has not given a date for results. The trial registry estimates the main data will be collected by January 2027, with the study finishing in September 2028.

The registry lists one recruiting site in Beijing. The FDA-cleared US trial has not been registered as recruiting that we could find. We found no expanded access programme.

Trials and programmes

• XS228CN Phase 1NCT07118319 · recruiting · Peking University Third Hospital, Beijing, China

Signal measures how often, and how reliably, XellSmart Bio-Pharmaceutical (Suzhou) tells the public about this drug. It says nothing about whether the drug works. A quiet company isn't necessarily a bad sign, but regular, on-time updates help patients plan.

70 or more is Active, 45 or more is Steady, 20 or more is Quiet, below 20 is Silent. Full method.

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