TECHNOLOGY EXPLAINER
Drug Discovery Modalities
— Two orders of magnitude in molecular weight and twelve in molecule count: drug types are also material types
Low」s, peptides, nucleic acids, antibodies, mRNA, gene therapy, cell therapy — the type of medicineHomeis called. weight isAbout 590"Number of grains" that enter the body one time10 to 10 ridesOpen The world is different for each modalities.
Concept diagram of AI generation- What is the drug discovery modalities (3 lines)
- Total Map - Compare the main modalities with four axis
- Compare weight: Approx. 590 to 150,000
- Material Technician Perspective 1: From Pure Material to Material with Distribution
- in the way of making - organic , solid phase , cells and cells themselves
- “40 Amino Acids” – Where is the regulation drawn?
- Dosage routes and target locations
- Calculation of this paper: "Number of grains" included in one time
- Material Technician Perspective 2: The technology to measure changes when the unit changes
- Counting new drugs in 2025 with modalities
- Issues and things that have not yet been determined
- Glossary/Reference Materials/Reference Table
Facts= Contents described in the publication material (with source link)
The calculation of this paper= The value calculated based on the specified premise
Undetermined / Future= Items that have not been confirmed as a plan and goal
In addition to this, it is possible to organize structures and read materials and process designs."Description"is distinguished.
About this articleExplanation of pharmaceutical products cross-sec from the viewpoint of “materials and manufacturing technologies”Home not to evaluate or compare the effects and safety of the treatment, No medical advice. The name of the product isExamples of weight, route and manufacturing methodsto quote the description of the attachment, There is no intention to recommend or inferior certain drugs or treatments. The number of doses is used as a material that converts "number of grains" as it is, and it is not a description of how to use it.
1. What is the drug discovery modalities (3 lines)
- What to say:modalityTypes of "things" to be put into the body as a medicineComment: Treatment with low-molecular compounds, peptides, oligonucleic acids, antibodies, mRNAs, and viral vectors, and cell therapy. (not legally defined words)
- What's different:The difference is not only the mechanism of action.Location size, how to make, how to put into the body, where to be deliveredBut the modalities vary greatly
- Why Materials Talk:Organic solvents and crystallization, solid phase resin, cell culture medium and chromatography carriers, lipids of lipid nanoparticles, bags that contain cells—When modalities change, the combination of the necessary materials and devices will be replaced.(This article)
The difference between modalities is easy to understand when reading as a difference in material hierarchy. Low-molecularSingle compoundpeptide and oligonucleic acidORIGOMERAntibodyHigh s with distribution of post-translation modificationHome TherapyViral particlesCell therapyLive CellsComment ICH’s quality guidelines are also used in chemical、 products (Q6A) and biotechnology applications (Q6B).Separate documentsHOME Q6B“Since bio、 process is used for the production of organisms, biological structural unevenness occurs in proteins.”I wroteFacts。
2. Total map - Compare the main modalities with four axis
First of all, the main modalities using the numbers written in the attachment of the approved productweight, how to make, route of administration, target4 axis. The details of each modalities are handled in the series (Peptides and GLP-1, Nucleic Acid Drugs, Antibody Drugs and ADCs, mRNA, viral vectors, CAR-T cells, and target protein decomposition). About this articleCross-sec comparisonFocus on
| Home | Example (Attachment) | weight, etc. | How to make | Dosage route | Target and action points |
|---|---|---|---|---|---|
| Low molecular | GLEEVEC（imatinib） | 589.7 g/) (mecilate) | Not specified in the attachment (chemical composite) | Oral (tablets) | Kinase (Inhibition) |
| Decomposition Inductor | VEPPANU（vepdegestrant） | 723.90 Da | Not specified in the attachment | Oral (tablets) | E gen receptorsDecompositionContact Us |
| Peptide | OZEMPIC（semaglutide） | 4113.58 g/mol | Peptide skeleton is yeast fermentationChemical Qualification Index | Subcutaneous injection (with oral tablets) | GLP-1 receptor (operation) |
| Antis Nucleic Acid | SPINRAZA（nusinersen） | 7501.0 Da (s) salt) | Not specified in the attachment | Intrathecal | SMN2 transfer product (RNA) |
| siRNA | ONPATTRO（patisiran） | 14304 Da (s) salt) | Lipid complexFormulation as | Infusion | TTR mRNA (RNA) |
| Antibody | HUMIRA（adalimumab） | Approx. 148 kDa (1330 amino acids) | CHO cellsRecombinant expression of production | Subcutaneous injection | TNF |
| Therapy (AAV) | ZOLGENSMA | Not weightGenomicsDisplay | Recombinant Self-Phase AAV9 | Infusion | Deliver genes to code SMN protein |
| Cell Therapy | KYMRIAH（tisagenlecleucel） | Not weightNumber of cellsDisplay | Patientification of the patient's own T cells with a virus vector | Infusion | Targeting CD19 |
AllFacts(Reference Material 11-19) "Not specified in the attached document" indicates that the document referenced by this article was not able to confirm the description of the manufacturing method. Since mRNA is handled in this series "mRNA" section, it is saved from the table.
3. Line by 590 weight: Approx. 590 to 150,000
Lipinski et al.」 of FiveComment The abstract of the paper in 2001 is"Hydrogen binding body exceeds 5; if the hydrogen binding receptor exceeds 10, the body weight exceeds 500, and the calculation LogP (CLogP) exceeds 5, absorption and membrane transmission is likely to be worse."Facts。
Where is the actual approved drug? Let's align the weight described in the attached document with the multiple scale.
Conversion from the description of the attachmentThe calculation of this paper。
- Antibody ÷ Low s:148,000 ÷ 589.7 ＝ Approx. 251 times(2.4 2.4 digits)
- Peptide ÷ Low s:4,113.58 ÷ 589.7 ＝ Approx. 7.0 times
- Antibody ÷ Peptide:148,000 ÷ 4,113.58 ＝ Approx. 36 times
- Decomposition Inductor:"723.90 of vepdegestrant is a rule of weight"More than 500 weightHomeOral TabletsApproved asFacts
Premises and Limits:"imatinib is a sodium salt weight of sodium and is not free. 148 kDa of antibody is the word "about"." Conversion to see differences in digitsand does not indicate the superiority of the drug.
4. Perspective of Material Technicians 1: From Pure Sub s to Materials with Distribution
ICH’s quality guidelines are the standard for chemical、 products.Q6A, Oct. 6, 1999 Step 4) and the standard for biotechnology applicationsQ6BMarch 10, 1999 Step 4)Different documentsFacts。
The description of Q6B is a familiar idea for engineers of polymer materials. "The target substance can be a mixture of predicted post-translation modifiers (e.g. sugar-chain modifiers)"Home and“Since the unevenness of these products defines its quality, it should analyze the degree of unevenness and profile and ensure consistency between lots.”Facts。
In short s"Purpose compound + impurities"The standard is formed in the viewpoint of In antibody and protein"Distribution itself is a product"It is a view. From single-dispersed low- compounds to polymers with weight distribution and composition distribution——I read that the standard idea is switched to it (in this article).
The concept of impurities changes. Q6B comes from impurities such as host cell proteins, host cell DNA, and medium components.Process-derived impuritiesHome Precursors and decompositions that occur during manufacturing and storage"Purpose material-derived impurities"Facts。 The material used for culture becomes the origin of impuritiesTherefore, the selection of materials such as medium, resin, and filter will be a quality story as it is (in this article).
and interestingMiddle AreaHOME Q6A"It may be applied to low weight peptides, but it is not enough to describe the standard of higher weight peptides, polypeptides, and biotechnology applications."HOME MoreOrigonucleotideNot applicableFacts。 It does not fit in the frame of low s to create by chemical——Peptides and oligonucleic acids are modalities in “between” above the guidelines (in this article).
5. in the way of making: organic , solid phase , cells and cells themselves
When the modalities change, the "type" of the manufacturing will change. In this section, we will organize it into four types within the scope that can be confirmed by the publication material.
(1) Solid Phase Synthesis Approach to Peptides and Oligonucleic Acids
1984 Nobel Chemical Award for R. Bruce Merrifield"Development of Methodology of Chemical Synthesis on Solid Phase"Awarded toFactsHome The first amino acid is combined into polymers,Derivatives and unresponsive starting substances after each process are removed by "filtration and cleaning of polymers"I explain how toFacts。
In addition, as an application of this idea“The most important thing is definitely the、 of oligonucleotides.”Contact UsFacts。 Peptides and oligonucleic acids are based on the concept of “coupling into solid phases and stretching one step by step”It is.
(2) Cell-based antibody and part of peptide
HUMIRA Attachments Adalimumab“Mammalian cells (Chinese hamster ovaries) are produced by recombinant DNA technology and refined in processes including certain viral inactivation and removal processes.”FactsHome On the other hand, there are examples of using cells in peptides. About semaglutide"Peptide skeleton is produced by yeast fermentation"Facts。 The production of peptides is not determined as chemical .Home The use of this product is handled by this series "peptide medicine and GLP-1".
(3) Live cells and virus particles themselves
KY AH"Consisting of self-identified T cells using wrench virus vector"FactsHome ZOLGENSMA"Recombinant Self-Phase AAV9"concentration2.0 × 1013 vg/mLHomeFactsHome Details of production are handled in this series "CAR-T cells" and "Virus vector".
6. “40 amino acids” boundary: Where is the regulation drawn?
The precincts of peptides and proteins are chemically continuous. However, U.S. regulationsClear LineYes.
“Protein is a alpha-amino acid polymer with a specific set array of sizes exceeding 40 amino acids.”Facts
MoreDetermine the size of the amino acids in those chains, not in consecutive arrays, if two or more chains are met in natural form.Facts。
This line is directly connected to practice. FDA, BPCI Law)」 to be a licenseBy the provision March 23, 2020The approval of 'drug formulation' which was approved in Article 505 of the FD&C ActBe as BLAI explain that it wasFactsHome For exampleInsulin and Insulin Analog, Human Growth Hormone, Pancreatic Enzyme, Reproductive HormoneOther “This amendment does not affect peptides (i.e., polymers consisting of amino acids below 40).”Facts。
| Line Pull | Either side | Source Description |
|---|---|---|
| US: Definition of protein | 41 amino acids or more = protein (a framework of biological preparation) | 21 CFR 600.3(h)(6) |
| USA: Peptide | Less than 40 amino acids = peptide (pharmaceu)s = drug) | FDA "Unauthorized" clause |
| ICH Q6A | Applicable to peptides with low weight. Insufficient peptides and polypeptides of high weight. Origonucleotides are not eligible | Q6A 1.3 |
| ICH Q6B | Proteins, polypeptides and their derivatives, and products that are composed of them (e.g. complexes) | Q6B 1.3 |
AllFacts(eCFR[Reference Material 3], FDA[Reference Material 4], ICH Q6A[Reference Material 5], ICH Q6B[Reference Material 6]) The。al standard of "40 amino acids" is based on the U.S. regulations, and it is not confirmed whether other countries and regions adopt the same standard.
Premise:Average mass of one amino acid residueAbout 110Close Assumption of this article. Actually depends on the array)The calculation of this paper。
- 40 x 110 = about 4,400
- semaglutide weight 4113.58, thisClose to 40 residuesContact Us
- adalimumab 1330 amino acids(Attachment)FactsHome BoundaryApprox. 33 timesLength 1330 ÷ 40)
Premises and Limits:semaglutide contains modifications such as fatty acid chains, so simple conversion from residual numbers is not applicable.The number of amino acids, not weightsContact Us
7. Dosage routes and target locations
When choosing modalities, you can’t detach “where to target” and “how to put in the body”.
(1) What kind of medicines have been used for?
Santos et al is a paper in 2017893 bio's acting by 1,578 drugs approved by the US FDAorganize andProtein targets667 from human genome are 667It is reported thatFactsHome For analysis1,419 low doses and 250 biological preparationsHomeFacts。
- 4 "privileged" families:Rodopsin G-protein cooperative receptor (GPCR), ion channel, protein kinase, and nuclear hormone receptors.44% of human protein targets(GPCR 12%, ion channel 19%, kinase 10%, nuclear receptor 3%Facts
- :ion to hypo: drugs:These 4 families70% of hypo drugs(GPCR 33%, ion channel 18%, kinase 3%, nuclear receptor 16%)Facts
- Biological formulation target:Publications“In biological formulations, toxic proteins and cell surface antigen proteins are the most important target class.”Facts
(2) Target location and means of delivery
(3) Dosage routes are determined by the nature of the
| Example | Dosage route | Read from the attachment |
|---|---|---|
| GLEEVEC | Oral (tablets) | MecilatepH soluble in water-based buffer liquid below 5.5, Neutral to alkaline to slightly dissolve or insoluble |
| VEPPANU | Oral (tablets) | dissolution is pH dependent,It is easy to dissolve in the stomach pH and slightly melts in the intestine pHHome E gen receptor binding sites and E3 ligase binding sites are connected with linkersLow」sContact Us |
| OZEMPIC | Subcutaneous injection | The main mechanism for weekly administrationAlbumin bindingHome Addition of hydrophilic spacer to 26th lysine and C18 fat diac acid |
| RY SUS・OZEMPIC Tablet | Oral (tablets) | Ab er SNACPreparation with salkaprosate sodium. absorption is mainlyStomachHome Estimated absolute bioavailabilityApprox. 0.4 to 1%（RYBELSUS） |
| SPINRAZA | Intrathecal | Injected into the cerebro nal fluid to the central nervous system |
| ONPATTRO | Infusion | siRNALipid complexFormulation and delivery to liver cells |
| HUMIRA | Subcutaneous injection | Solution with glass prefilled syringe.No preservatives |
AllFacts(Reference Material 11-17) This table is aligned with the description of formulation and properties.Not a description of how to use。
peptides and antibodies are used for low s where oral administration is commonBasic injectionfor oralizationAnother material called absorberrequired. Estimated absolute bioavailability even with its oral semaglutideApprox. 0.4 to 1%HomeFacts。 The larger the , the more “to put in the body” itself becomes a material design issue——This detail is handled by the series “Peptides and GLP-1”, “DDS and Targeting” and “Blood Brain Gate”.
8. Calculation in this article: "Number of grains" included in one time
The difference between modalities is another when you look at the dose unit of the attachment. Low, peptides, nucleic acids and antibodiesmg (mass)AAV gene therapyvg/kgCAR-TNumber of cellswritten inFactsHome Let’s convert the weight to the number of s and put it on the same thing.
- GLEEVEC：400 mg tablets mecilate 478 mg include: Mecilate's weight 589.7Facts
- OZEMPIC：One of the dosage descriptions 1 mgmolecular weight 4113.58Facts
- SPINRAZA：12 mg/5 mL preparation nusinersen sodium salt per mL 2.53 mg(12.65 mg at 5 mL). Weight 7501.0Facts
- HUMIRA：One of the dosage descriptions 40 mgquantity weight 148 kDaFacts
- ZOLGENSMA：1.1 × 1014 vg/kgFacts
- KYMRIAH：Adult large cell type B-cell lymphoma etc. 0.6〜6.0 × 108 CAR-positive survival T cellsFacts
Avocado Number 6.022 × 1023 Convert /The calculation of this paper。
- imatinib：0.478 g ÷ 589.7 ＝ 8.11 × 10-4 mol → Approx. 4.9 × 1020
- nusinersen：0.01265 g ÷ 7501.0 ＝ 1.69 × 10-6 mol → About 1.0 × 1018
- semaglutide：0.001 g ÷ 4113.58 ＝ 2.43 × 10-7 mol → About 1.5 × 1017
- adalimumab：0.040 g ÷ 148,000 ＝ 2.70 × 10-7 mol → Approx. 1.6 × 1017
- ZOLGENSMA：Weight per kg 1.1 × 1014 vector genomics
- KYMRIAH：108HomeThe cells of the attached document themselves
Most imatinib (approx. 4.9 × 10)20) and the fewest KY AH (0.6 × 10)812 digits20 ÷ 0.6 × 108 ≈ 8 × 1012）。
Premises and Limits:Dosage of the values listed in the attachmentExampleIt depends on dosage interval, weight, and adaptation. ZOLGENSMA is a value per kg of weight and does not include other conditions. Less than one dose does not indicate the strength or superiority of the drug.This is a conversion to indicate that the counting unit changes with modalities.
9. Material Technician Perspective 2: When the unit changes, the measurement technology changes
The conversion of Chapter 8 shows:There is a world that can be managed by mass and a world that must be managed by the number ofThat is This article explains.
- The world written by mg:Low, peptide, nucleic acid and antibody. Chemical notificationchromatography and spectroscopycan
- World of VG:AAV. ZOLGENSMA「2.0 × 1013 vg/mL」HomeNumber of genomesFacts
- World of Cell Number:CAR-T KY AH Attachments Dose"Number of CAR-positive survival T cells"I am determined to beFacts。“Do you live?” “Do you express CAR?”You need to include up to
Subst、tion to the world of materialsMigration from solution concentration management to particle count and particle size distribution managementClose Change This article explains. For engineers who have handled particle count and distributed stability,Analysis of viral vectors and cell preparations is rather a field with land accountsComment
Another in intentContainers and liquid materialsComment 102010 tablets with individual s8In bags containing cells, weight of 'one piece' that the wall of the container or breaks by the weightis completely different. Less modality,Adsorption, e tion, and shearing of liquid materials can affect the quantity of products.It is considered (explanation by this article). The requirements for the liquid contact material are handled in detail in this series "GMP".
10. Count the new drug in 2025 with modalities
FDA Pharmaceutical Evaluation Research Center (CDER)Approved 46 new drugs (novel drugs) in 2025HomeFactsHome This list is attached to the general name ending (stem) and biological formulation4-character suffixLet's separate with or without.
This article was classified as follows from the general name of the FDA list [Reference Material 7]The calculation of this paper。
- Antibody and other proteins (with 4-character suffix): 12 items(About 26 items —— 10 items of -mab (narsoplimab, depemokimab, sibeprenlimab, linvolamab, garadacimab, clesrovimab, telisotuzumab vedotin, nipocalimab, penpulimab, datopotamab deruxtecan. ADC), pembrolizumab and enzymatic berahyaluronidase alfa are combined with vedotin and deruxtecan.
- oligonucleic acid(about 7%)——zazasiran, fitusiran, donidalorsen
- Peptide: 1 itemelamipretide
- Low items: 30 items(about 65——other than the above)
Premises and Limits:The classification is based on the ending and suffix of the general nameJudgmentIt is not the classification indicated by the FDA. This list isCDER approval onlyCell therapy and gene therapy (CBER) are not included. It is a number of a year and does not indicate a trend.
On the side of CBER, as of September 17, 2026, the list of "Approved Cells and Therapy Products"54 Products(The number of lines in the list is counted)The calculation of this paperHome In addition to gene therapy such as CAR-T cells and AAV,Hematopoietic cell products from cord bloodHomeCell products cultured on collagen filmAlso includedFacts。
Target proteinDissolve rather than in tingThe way of thinking of s is Sakamoto in 2001「Protac（proteolysis-targeting chimeric molecule）」as reported. The target protein is artificially pulled into the biotin ligase complex, and then decomposed.ICH Q6A is not eligible for oligonucleotides and is insufficient for high-weight peptides.It is said that the chimera compound is synthesizedFacts。
FDAMay 1, 2026、vepdegestrant（VEPPANU）approve and"Hetero dual-sensitive protein decomposition derivative"FactsHome From reporting to approval25 yearsHomeThe calculation of this paperHome This article does not include "the first PROTAC approval" on the FDA approval pageFDA Expression OnlyQuote Details are handled by this series "targeted protein decomposition".
11. Challenges and things that have not yet been determined
(1) The standard of boundary modalities is still on the rise
About 65% of CDER new drugs in 2025 were low-weight drugs.FactsHome How the internationally harmonized standards will be organized on the modalities of such “between”?I couldn't confirm the scope of this article.Undetermined / Future。
(2) The future of modalities is not determined by public primary information
(3) Outside of the low-‘rules’The calculation of this paperbut thisYearComment I could not confirm the future composition ratio of modalities by primary information such as regulatory authorities.The market estimate of the survey company is not used by this series.Undetermined / Future
is an example that indicates there is an oral area outside the rule of bu.
weight 723.90 decomposition inductors have been approved as oral tabletsFactsHowever, the rules of Lipinski are"It is likely that the absorption and membrane transmission will become worse"It is a probability ofFacts。 The primary information that can be generalized to what extent can be spread outside was not confirmed in the scope of this article(This article)
- The modalities are the type of medicine and the hierarchy of materials.Single compound → Array oligomer → High-molecular drugs with distribution → Particles and cells (arrangement as described in this article)
- weight is about 590 to 15 million.Antibody is about 250 times the lowThe calculation of this paper
- ICH’s quality guidelines are also divided by chemical composites (Q6A) and bio-products (Q6B).Q6B defines quality of unevenness itselfFacts
- In the U.S., "over 40 amino acids" are proteins.Regulatory framework changes with peptides and proteinsFacts
- The number of grains per serving is 1020(Low)) to 10812 digits open until (cell)The calculation of this paper
- Of 46 CDER new drugs in 2025, low、s were 30 itemsThe calculation of this paper
12. Glossary
- Home
- Types of “things” that are put into the body as pharmaceuticals. Low。s, peptides, nucleic acids, antibodies, cells, etc.
- Low-weight drugs
- Medi s of hundreds of s made of organic . Many are used orally.
- of Five
- Lipinski et al., 。 weight 500 or more.
- Peptide
- High-molecular drugs with amino acids. In U.S. regulations, less than 40 amino acids are peptides.
- oligonucleic acid
- short nucleic acid drugs. Antis (ASO) and siRNA target RNA.
- Antibodies
- Antibodies (proteins) drugs that bind to specific opponents.。 weight is about 150,000.
- Protein degradation in dector
- A molecule that causes the target protein to be degraded and degraded.
- Therapy
- Treating genes to cells. Virus vectors such as AAV are used.
- Cell Therapy
- Treatment of living cells. CAR-T cells, etc.
- vg (Vector Genome)
- Number of genes that the virus vector carries. AAV product concentration and dose unit.
- BLA
- U.S. biological formulation approval application. Different framework from NDA of chemical drugs.
- CDER／CBER
- FDA Pharmaceutical Evaluation Research Center / Biological Formulation Evaluation Research Center.
- ICH Q6A／Q6B
- International guidelines for pharmaceutical standards. Q6A is a chemical composite product, Q6B is a bio product.
- Unevenness
- In the same product, there is a mixture of modifications with different modifications. Quality in Bio Products
- Solid Phase Syn
- Synthetic methods that combine s into solid polymers and stretch them one by one. Can be cleaned.
- Bioavailability
- The rate of blood flow of the whole body of the dose.
13. Reference materials (primary information)
- Santos「A comprehensive map of molecular drug targets」Nature Reviews Drug Discovery 16(1), 19–34 (2017). doi:10.1038/nrd.2016.230 — pmc.ncbi.nlm.nih.gov
- Lipinski, C.A., Lombardo, F., Dominy, B.W., Feeney, P.J.「Experimental and computational approaches to estimate solubility and permeability in drug discovery and development settings」Advanced Drug Delivery Reviews 46(1–3), 3–26 (2001). doi:10.1016/S0169-409X(00)00129-0 — pubmed.ncbi.nlm.nih.gov
- United States Federal Regulations (eCFR)「21 CFR 600.3 Definitions」 — ecfr.gov
- FDA「"Deemed to be a License" Provision of the BPCI Act」 — fda.gov
- ICHQ6A: Specifications: Test Procedures and Acceptance Criteria for New Drug Sub)s and New Drug Products: Chemical Sub)s database.ich.org
- ICHQ6B: Specifications: Test Procedures and Acceptance Criteria for Biotechnological/Biological Products database.ich.org
- FDA「Novel Drug Approvals for 2025」 — fda.gov
- FDA「Approved Cellular and Gene Therapy Products」 — fda.gov
- FDA"FDA approves vepdegestrant for ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer" May 1, 2026 — fda.gov
- Sakamoto「Protacs: chimeric molecules that target proteins to the Skp1-Cullin-F box complex for ubiquitination and degradation」PNAS 98(15), 8554–8559 (2001). doi:10.1073/pnas.141230798 — pubmed.ncbi.nlm.nih.gov
- DailyMed「GLEEVEC (imatinib mesylate) tablets」 — dailymed.nlm.nih.gov
- DailyMed「VEPPANU (vepdegestrant) tablets」 — dailymed.nlm.nih.gov
- DailyMed「OZEMPIC (semaglutide) injection」 — dailymed.nlm.nih.gov
- DailyMed「RYBELSUS／OZEMPIC (semaglutide) tablets」 — dailymed.nlm.nih.gov
- DailyMed「SPINRAZA (nusinersen) injection」 — dailymed.nlm.nih.gov
- DailyMed「ONPATTRO (patisiran) lipid complex injection」 — dailymed.nlm.nih.gov
- DailyMed「HUMIRA (adalimumab) injection」 — dailymed.nlm.nih.gov
- DailyMed「ZOLGENSMA (onasemnogene abeparvovec-xioi) suspension」 — dailymed.nlm.nih.gov
- DailyMed「KYMRIAH (tisagenlecleucel) suspension」 — dailymed.nlm.nih.gov
- The Royal Swedish Academy of Sciences「The Nobel Prize in Chemistry 1984 — Press release」 — nobelprize.org
14. Response Table of Claim and Sources (Audit)
| Content | Home | |
|---|---|---|
| The FDA-approved drug of 1,578 organizes 893 bio。es, and the protein target of human genomes is 667. The analysis target is 1,419 low s and 250 biological preparations. GPCR, ion channel, kinase, and nuclear receptors account for 44% (12%, 19%, 10%, 3%) of human protein targets, and are responsible for the effect of 70% (33%, 18%, 3%, and 16。 of low-。 drugs. Biological formulations are the most important target class of toxic proteins and cell surface antigens | Santos et al (2017)[Reference 1]https://pmc.ncbi.nlm.nih.gov/articles/PMC6314433/ | Facts |
| "Rules of Five" which is likely to be absorbed and membrane transmission worse if the receptor exceeds 10, body weight exceeds 500, CLogP exceeds 5 | Lipinski et al (2001)[Reference 2]https://pubmed.ncbi.nlm.nih.gov/11259830/ | Facts |
| The protein is defined as a alpha-amino acid polymer with a specific set array of sizes exceeding 40 amino acids, and it is determined by the total number of amino acids in the chain where multiple chains are seen naturally. | 21 CFR 600.3(h)(6)[Reference 3]https://www.ecfr.gov/current/title-21/chapter-I/subchapter-F/part-600/subpart-A/section-600.3 | Facts |
| On March 23, 2020, the approval of biological preparations approved in Article 505 of the FD&C Act was considered BLA. Insulin and insulin analogues, human growth hormone, pancreatic。s, reproductive hormones, etc. This amendment does not affect peptides consisting of 40 or less amino acids | FDA "Unauthorized" clause[Reference 4]https://www.fda.gov/drugs/guidance-compliance-regulatory-information/deemed-be-license-provision-bpci-act | Facts |
| Q6A Step 4 is October 6, 1999. Q6A may be applied to low-weight peptides, but it is not sufficient for high-weight peptides, polypeptides and bioproducts, or oligonucleotides, etc. | ICH Q6A[Reference 5]https://database.ich.org/sites/default/files/Q6A%20Guideline.pdf | Facts |
| Q6B’s Step 4 is March 10, 1999. A product (e.g., a complex) that is composed of protein, polypeptide and its derivatives. In the biosyn process, biological structural unevenness is generated in proteins, and the object is a mixture of post-translation modifiers (e.g. sugar-chain modifiers), and the degree and profile should be analyzed to ensure consistency between lots. Process-derived impurities (home cell proteins, host cell DNA, medium components, etc.) and object-derived impurities (precursors, decompositions, etc.) | ICH Q6B[Reference Material 6]https://database.ich.org/sites/default/files/Q6B%20Guideline.pdf | Facts |
| CDER approved 46 new drugs in 2025, and the general names of each item (narsoplimab-wuug, depemokimab-ulaa, lerodalcibep-liga, sibeprenlimab-szsi, pembrolizumab and berahyaluronidase alfa-pmph, linvo tamab-gcpt, garadacimab-gxii, clesrovimab-cfor, telisotuzumab-liga, sibrandomina, s, sibfits, szsi, szsi, szszsi, sam, szsi, szsi, s, szsi, szsi, s, szsi, s, szsi, szsi, s, s, s, szsi, szsi, szsi, etc. | FDA「Novel Drug Approvals for 2025」[Reference Material 7]https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2025 | Facts |
| The list of approved FDA cells and gene therapy products includes gene therapy such as CAR-T cells, AAVs, blood cell products from umbilical cords and cell products cultured on collagen membranes (updated on September 17, 2026) | FDA「Approved Cellular and Gene Therapy Products」[Reference 8]https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products | Facts |
| The FDA approves vepdegestrant (VEPPANU) on May 1, 2026 and describes it as heterogeneous protein decomposition derivative | FDA approval page[Reference 9]https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-vepdegestrant-er-positive-her2-negative-esr1-mutated-advanced-or-metastatic-breast | Facts |
| Protac-1 is synthesized as a chimera compound that attracts target proteins to the ubiquitin ligase complex SCF, causing ubiquitination and degradation (2001) | Sakamoto et al (2001)[Reference Materials 10]https://pubmed.ncbi.nlm.nih.gov/11438690/ | Facts |
| imatinib is a kinase in tor and oral tablet, that the molecular weight of methylates is 589.7 g/mol, that 400 mg tablets contain 478 mg methylate, that methylates are soluble in water-based buffers of pH 5.5 or less, and have a 400 mg/day dosage description | EEVEC[Reference Materials 11]https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=211ef2da-2868-4a77-8055-1cb2cd78e24b | Facts |
| vepdegestrant is a low- heterogeneous protein decomposition derivative that connects estrogen receptor binding sites and E3-ligase binding sites with a linker, the ve weight is 723.90 Da, the soluble level is pH-dependent, easy to dissolve in the stomach pH, slightly soluble in the intestinal pH, oral tablets | VEPPANU[Reference Material 12]https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=484e21ba-8fe1-4c6d-b923-8ba4cf5b2f20 | Facts |
| semaglutide’s peptide skeleton is produced in yeast fermentation, the main mechanism is a hydrophilic trans to 26th place lysine and the addition of C18 fatty acid to albutin binding, that the molecular weight is 4113.58 g/mol, that it is subcutaneous injection, that there is 1 mg/week in the dosage description, is a GLP-1 receptor agonist | OZEMPIC[Reference 13]https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=adec4fd2-6858-4c99-91d4-531f5f2a2d79 | Facts |
| Oral semaglutin tablets are formulated with SNAC (Salkaprosato Sodium), and the absorption is mainly caused by the stomach, the RY absolute bioavailability is about 0.4-1% with RY SUS | RY SUS/OZEMPIC[Reference 14]https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=27f15fac-7d98-4114-a2ec-92494a91da98 | Facts |
| nusinersen is administered in the marrow cavity with antisense oligonucleotide targeting SMN2, distributed from the cerebrospinal fluid to the central nervous system, molecular weight is 7501.0 Da (salt), 12 mg/5 mL preparation contains 2.53 mg sodium salt per mL | SPINRAZA[Reference Materials 15]https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=dd70cd5f-b0fc-4ba4-a5ea-89a34778bd94 | Facts |
| patisiran is a two-stranded siRNA, which is not formulated as a lipid complex that can be delivered to hepatocellular cells, and is combined into a 3′ non-translation region of TTR mRNA, the si weight of sodium salt is 14304 Da, and drip static injection. | ONPATTRO[Reference 16]https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=e87ec36f-b4b4-49d4-aea4-d4ffb09b0970 | Facts |
| adalimumab is a TNF in tor that is produced by recombinant DNA technology in the CHO cell expression system and is purified in the process including the viral inactivation and removal process, 1330 amino acids and 148 weight of 148 kDa, a solution that does not contain preservatives of subcutaneous injections, is filled with glass prefilled syringes, with 40 mg biweeks in the dosage description | HUMIRA[Reference Materials 17]https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=608d4f0d-b19f-46d3-749a-7159aa5f933d | Facts |
| ZOLGENSMA contains an introduction gene that codes SMN protein in recombinant self-phase AAV9, the no concentration is 2.0 × 10^13 vg/mL, the recommended dose is 1.1 × 10^14 vg/kg, the drip static injection | ZOLGENSMA[Reference Materials 18]https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=68cd4f06-70e1-40d8-bedb-609ec0afa471 | Facts |
| KY AH is composed of a self-house T-cell that introduced anti-CD19 CAR with a lentivirus vector, that the dose is based on the number of CAR-positive survival T-cells, 0.6 to 6.0 × 10^8 cells such as adult mantle large cell type B-cell lymphoma, and drip static injection. | KY AH[Reference Materials 19]https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=aad3ba54-dfd3-4cb3-9e2b-c5ef89559189 | Facts |
| The 1984 Novel Chemical Award was awarded to the methodology of chemical on the solid phase of Merrifield, which is the method to combine the first amino acid into polymers to remove the byproducts after each process by filtration and cleaning, and the most important application of this idea is the synthesis of oligonucleotide. | Nobel Prize 1984 Press Release[Reference Materials 20]https://www.nobelprize.org/prizes/chemistry/1984/press-release/ | Facts |
| The ratio of the weight (approximately 251 times, approximately 7.0 times, approximately 36 times) and vepdegestrant is about 1.4 times the weight 500. 40 residue x 110 = about 4,400, 1330÷ 40 = about 33 times. The number of 4.9s (imatinib approximately 4.9×10^20, nusinersen approximately 1.0×10^18, semaglutide approximately 1.5×10^17, adalimumab approximately 1.6×10^17) and approximately 12 digits difference. Classification of 46 CDER new drugs in 2025 (low-molecular 30, antibodies, etc. 12, nucleic acid 3, peptide 1) and rate of action. Number of 54 products of cell and gene therapy products. 25 years from Protac reporting to approval. | The calculation of this article. The average mass of amino acid residue is 110, avocadolo number 6.22×10^23, using the salt weight of the form as it is, using the example of the value described as a dose is the premise. The classification of modalities is not a FDA classification in the judgment of this article based on the general name ending and suffix. The number does not indicate the strength or superiority of the drug. | The calculation of this paper |
| How will the international harmony of boundary modalities (oligonucleic acids and polymeric peptides) be organized in the future? modalities different future composition ratio | At the time of the investigation of this article (September 2026), the primary information such as the regulatory authorities indicating this could not be confirmed. Market estimates of survey companies are not used | Undetermined / Future |
| vepdegestrant is 'first PROTAC approval'. The '40 amino acids' standard is adopted by non-US. Can generalize the spread of oral medicine to the outside of the rule of thumb? | The FDA’s approval page does not contain “first” and does not include other things because the primary information cannot be confirmed within the scope of the survey. | |
| Drug modalities as a material hierarchy (single compounds, array oligomers, polymers, particles and cells). Read that the standard idea is to switch from “pure substance” to “combination”. Peptides and oligonucleic acids are positioned as modalities between them. Examples of materials of the main role are divided into four types of manufacturing. The location of the target was organized in concentric circle. Read that the transition from "quantity" to "counting", the impact of container absorption and shearing of containers and liquid materials is as large as modalities with fewer amounts. The description of mRNA IVT refers to the contents of this series "mRNA" | and commentary of this article based on the publication content. It is not the opinion of authors and regulatory authorities of each paper | |
| Effectiveness and effect of each product, clinical results, safety and superiority of products | This article is a cross-disciplinary explanation of materials and manufacturing technologies, and does not evaluate and compare the efficacy and safety. The product name is quoted as an example of the weight, route, and manufacturing method. | |
| Fig. 1 - Fig. 5, Fig. 7 must be a diagram for explanation, not actual。s, cells, and devices. Hero image and figure 6 must be an AI generation image | Notes by this article |
Last Updated: September 26, 2026/Source is limited to primary information (DailyMed), FDA published materials, U.S. federal rules, ICH guidelines, and Nobel Foundation announcements. They are distinguished from the facts that have been sourced as "instructions" because they contain readings in material and process design. The future composition ratio of different modalities, the future of the boundary modalities, is not stated because it was not possible to confirm whether the first PROTAC was published. This article explains materials and manufacturing technologies.It does not evaluate and compare the effectiveness and safety of the treatment, and it is not a medical advice. All diagrams are illustrations for explanation. Fig. 1 - Fig. 5, Fig. 7 is an image of vector drawing, and Fig. 6 is an image of AI generation.We do not show actual s, particles, cells and products.