ARROWHEAD PHARMA

InvesterJones

bostonerimus said:

kinger101 said:

Your description of how RNAi works is wrong.  The API is RNA, not a protein.

Futhermore, there is no inherent targeting mechanism with RNAi.

And side effects are possible both from the API and other ingredients in the formulation.

Agreed, but careful with the acronyms. API in this context presumably means Advanced Pharmaceutical Ingredient, but I have heard some people on the "computer science" side of molecular biology describe RNA/DNA as being like an API (Advanced Programing Language) that instructs genes on how to make proteins. RNAi interferes with the manufacturing of proteins. To do that it must get into the cell and that is facilitated by attaching the RNA to a ligand that allows the whole molecule to cross the cell membrane. Investing in small companies in technical fields is dangerous for the regular investor as they don't have the technical expertise to really critique the company and it's assertions. Theranous is an extreme example, but working at the edge of a scientific field has many other risks both technical and regulatory. It's a field where specialized venture capital likes to work, but they will often get it wrong as well.

In biotech

API = Active Pharmaceutical Ingredient

In computer science

API = Application Programming Interface

kinger101

bostonerimus said:

kinger101 said:

Your description of how RNAi works is wrong.  The API is RNA, not a protein.

Futhermore, there is no inherent targeting mechanism with RNAi.

And side effects are possible both from the API and other ingredients in the formulation.

Agreed, but careful with the acronyms. API in this context presumably means Advanced Pharmaceutical Ingredient, but I have heard some people on the "computer science" side of molecular biology describe RNA/DNA as being like an API (Advanced Programing Language) that instructs genes on how to make proteins. RNAi interferes with the manufacturing of proteins. To do that it must get into the cell and that is facilitated by attaching the RNA to a ligand that allows the whole molecule to cross the cell membrane. Investing in small companies in technical fields is dangerous for the regular investor as they don't have the technical expertise to really critique the company and it's assertions. Theranous is an extreme example, but working at the edge of a scientific field has many other risks both technical and regulatory. It's a field where specialized venture capital likes to work, but they will often get it wrong as well.

API is Active Pharmaceutical Ingredient.  

The main advantage as I see it of RNAi and gene editing over small molecules is that not every gene or associated protein can be targeted by a small molecule.  This concept is know as drug-ability.   With DNA or RNA based therapies, this no longer applies.  

Re targeting to cells - use of ligands is not the only approach.  It is however the major hurdle to getting a macromolecule to work compared to a small molecule for many diseases.

I'm confused as to why computer scientists abbreviate "Language" with an "I", though.  I suppose after coding for a few years in C++, they may forget English.

InvesterJones

I'm confused as to why computer scientists abbreviate "Language" with an "I", though.  I suppose after coding for a few years in C++, they may forget English.

They don't. Closest people get to confusion is when they refer to 0 as the first of something But then again English speakers have been doing the same for years - which floor is the first floor again?

kinger101

InvesterJones said:

bostonerimus said:

kinger101 said:

Your description of how RNAi works is wrong.  The API is RNA, not a protein.

Futhermore, there is no inherent targeting mechanism with RNAi.

And side effects are possible both from the API and other ingredients in the formulation.

Agreed, but careful with the acronyms. API in this context presumably means Advanced Pharmaceutical Ingredient, but I have heard some people on the "computer science" side of molecular biology describe RNA/DNA as being like an API (Advanced Programing Language) that instructs genes on how to make proteins. RNAi interferes with the manufacturing of proteins. To do that it must get into the cell and that is facilitated by attaching the RNA to a ligand that allows the whole molecule to cross the cell membrane. Investing in small companies in technical fields is dangerous for the regular investor as they don't have the technical expertise to really critique the company and it's assertions. Theranous is an extreme example, but working at the edge of a scientific field has many other risks both technical and regulatory. It's a field where specialized venture capital likes to work, but they will often get it wrong as well.

In computer science

API = Application Programming Interface

 Isn't that a keyboard?

bostonerimus

InvesterJones said:

bostonerimus said:

kinger101 said:

Your description of how RNAi works is wrong.  The API is RNA, not a protein.

Futhermore, there is no inherent targeting mechanism with RNAi.

And side effects are possible both from the API and other ingredients in the formulation.

Agreed, but careful with the acronyms. API in this context presumably means Advanced Pharmaceutical Ingredient, but I have heard some people on the "computer science" side of molecular biology describe RNA/DNA as being like an API (Advanced Programing Language) that instructs genes on how to make proteins. RNAi interferes with the manufacturing of proteins. To do that it must get into the cell and that is facilitated by attaching the RNA to a ligand that allows the whole molecule to cross the cell membrane. Investing in small companies in technical fields is dangerous for the regular investor as they don't have the technical expertise to really critique the company and it's assertions. Theranous is an extreme example, but working at the edge of a scientific field has many other risks both technical and regulatory. It's a field where specialized venture capital likes to work, but they will often get it wrong as well.

In biotech

API = Active Pharmaceutical Ingredient

In computer science

API = Application Programming Interface

Yes, I got that wrong. The critical thing is the comparison of RNA/DNA to a set of commands to make things happen.

InvesterJones

bostonerimus said:

InvesterJones said:

bostonerimus said:

kinger101 said:

Your description of how RNAi works is wrong.  The API is RNA, not a protein.

Futhermore, there is no inherent targeting mechanism with RNAi.

And side effects are possible both from the API and other ingredients in the formulation.

Agreed, but careful with the acronyms. API in this context presumably means Advanced Pharmaceutical Ingredient, but I have heard some people on the "computer science" side of molecular biology describe RNA/DNA as being like an API (Advanced Programing Language) that instructs genes on how to make proteins. RNAi interferes with the manufacturing of proteins. To do that it must get into the cell and that is facilitated by attaching the RNA to a ligand that allows the whole molecule to cross the cell membrane. Investing in small companies in technical fields is dangerous for the regular investor as they don't have the technical expertise to really critique the company and it's assertions. Theranous is an extreme example, but working at the edge of a scientific field has many other risks both technical and regulatory. It's a field where specialized venture capital likes to work, but they will often get it wrong as well.

In biotech

API = Active Pharmaceutical Ingredient

In computer science

API = Application Programming Interface

Yes, I got that wrong. The critical thing is the comparison of RNA/DNA to a set of commands to make things happen.

Not a great comparison IMHO. It's the interface word in (computer science) API that's key to what they are, not the instruction set. In biological terms API is more like a surface protein than DNA/RNA.

BrockStoker

kinger101 said:

Your description of how RNAi works is wrong.  The API is RNA, not a protein.

Appreciate the feedback as always kinger.

Yes, you are right, RNA is not a protein. A bit of a brain fart there. However, it is essentially an organic molecule, made up of nucleotide bases. The point is, it's not likely to be harmful, and is quickly broken down.

Can you point out where I'm wrong about how RNAi works? Thanks.

My understanding is simply this: The API is delivered to the target tissue/organ due to the design of another part of the molecule.At the target tissue, the API triggers the genetic switch for a specific gene or genes, thereby turning off or moderating how much of a specific (troublesome) protein is produced.

kinger101 said:

Futhermore, there is no inherent targeting mechanism with RNAi.

Well, I didn't actually say that, and if I in anyway implied that, I did not mean to.

Indeed, delivering the API (thanks for reminding me of that term) to the correct tissue/organ is one of the toughest aspects to get right, especially when the target is not the liver.

That said, I believe it could be argued that RNAi molecules tend to end up in the liver, so the "default" target is liver tissue, even if that is not necessarily the intended target.

kinger101 said:

And side effects are possible both from the API and other ingredients in the formulation.

This may be true, but my understanding is that initial side effects are usually very minor (assuming the targeting is precise), and long term side effects are unlikely due to the nature of RNAi, as I explained above.