Foro BIOTECHNOLOGY ASSETS | Comentarios de BST -Pagina: 2233

Mensaje de: clayton Hora: 26/03/2026 10:06:39
Esto es Zip: Si la tecnología Splittera® se consolida como el estándar para el ensamblaje de genes e..........

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"Cobraría royalties", como dice gemini en este caso la empresa pasaría de definición de "joya en su nicho de mercado" a ser "unicornio" y solo teneis que buscar que es un inicornio en terminos de mercado

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26/03/2026 10:09:15

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Mensaje de: clayton Hora: 26/03/2026 10:06:39
Esto es Zip: Si la tecnología Splittera® se consolida como el estándar para el ensamblaje de genes e..........

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Fíjate solo los programas de Splicebio que usan ya Splittera, eran 23 diferentes. Solo con uno, tienes ingresos de 30-40 millones, más recurrencia en royalties de 5-10 anuales, solo por uno. Y esto es solo de Splicebio, recordemos que tenemos licencia con dos americanas, una cotizada. CAR-T y el resto de tecnología todavía sin licenciarse... es una broma que siga aquí, pero ellos deciden cuando hacer publi

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clayton

Pre

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26/03/2026 10:10:19

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Y esto no es calentar simplemente es la empresa en la que tenemos nuestro dinero.

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26/03/2026 10:12:36

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Por cada programa, Upfront de 0,5M a 10M€ y milestones entre 15-60M€, por cada programa. Y Splicebio acaba de pasar a Fase 2 de eficiencia con nuestra tecnología incorporada

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26/03/2026 10:14:51

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Solo Splicebio tiene 20 targets validados (podéis verlo en el vídeo), con 2-3 programas, solo de esta pata, 60 kilos fácil y si uno alcanza al mercado, unos 6-15M al año de recurrencia. Y Roche no pone 120 kilos sobre humo. Si la dejan cotizar, será una de esas "a ver si se hace un Solaria"... será un "a ver si se hace un BST"

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26/03/2026 10:16:29

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https://www.youtube.com/watch?v=syCL7T_qBBo&t=18s

os lo vuelvo a dejar, es largo, está en inglés, pero...

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26/03/2026 10:18:22

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Mensaje de: jonhlucas70 Hora: 26/03/2026 10:16:29

Thank you everyone. I"m Gerard. I"m the chief business officer at Splice Bio. We are a genetic medicines company mostly working in ofthalmology, but we"ve recently generated some exciting data in nonhuman primates in the CNS of nonhuman primates and that"s what I"ll be focusing on. uh today. So we have a novel technology in the genetic medicine space that essentially allows us to deliver genes that exceed the packaging capacity of AV vectors. That"s a major limitation in the gene therapy space where you cannot package uh the cargo that you like to deliver just because the cargo is too large and the vector is too small.

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26/03/2026 10:18:48

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Mensaje de: jonhlucas70 Hora: 26/03/2026 10:16:29

And we get around that by delivering a dual AAV that is uh superefficient compared to prior uh approaches. Our LEAP program as I said it is in the clinic. It"s a dual AB in stagger disease first inclass uh gene therapy completed the phase one part of the phase one two and now it"s enrolling patients in the efficacy part of the study those expansion randomized uh and adequately controlled. It"s supported by an observational uh clinical trial that we"ve been running uh for some years uh now and we have emerging pipeline of programs in of themology and CNS with validation across more than 20 therapy targets and validation in the writing and CNS in nonhuman primates which I"ll show uh some of it in here and obviously uh robust and strong uh IP position across platform as well as pr

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26/03/2026 10:19:35

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Mensaje de: jonhlucas70 Hora: 26/03/2026 10:16:29

We"ve raised uh over $200 million uh until now in in series A and series B uh financing. We have EQ, Sanopi and RO that participated in the series B as well as uh uh NEA, UCB, EOS, Novartis, Gilda and Asavis from the series A and this is the team that is leading the company. uh I am the chief business officer and have been since 2016. And this is the main challenge that we are addressing right in the genetic medicine space. The limited packaging capacity of these viral vectors that essentially precludes you from doing gene therapy uh for many of the targets that are actually interesting. There"s been several people trying to solve this problem before with other dual a technologies. They there are mostly three of them, right?

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26/03/2026 10:19:45

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Mensaje de: JAVIFM Hora: 26/03/2026 7:01:14
Good morning

mientras calientan, EL PAPELAZO APARECE = 0,24 = RESISTENCIA = VENTA

0,2360 1 030
0,2370 12 000
0,2390 27 445
0,2270 0,2400

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26/03/2026 10:19:59

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Mensaje de: jonhlucas70 Hora: 26/03/2026 10:16:29

And within re combination at the protein level, there is the first generation protein splicing approach using older versions of the technology. And then there is the third generation protein splicing, which is the one that we"ve been uh pioneering since we discovered protein splicing in the "9s. and we understood the limitations and challenges of first generation approaches. Now the way the technology works is that when you have a gene that is too large to be packaged in a in a in a single vector we can split that gene into halves. Uh to each of these halves we add these sequences encoding for third generation in which are protein domains that catalyze this protein splicing 3:26 - 4:05 reaction. Right?

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26/03/2026 10:20:34

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Mensaje de: jonhlucas70 Hora: 26/03/2026 10:16:29

So splicing but at the protein level and then we package each of these uh half trans genes in a separate AV vector. This dual AV vector transduces the cell and then you have expression and translation of each of these halves and it is at the and it is at the protein level that these uh intenses will find one another. they will form a non uh covealent uh protein protein interaction that will trigger the protein splicing reaction whereby they will essentially liate the last amino acid of the first fragment with the first amino acid of the second fragment and reconstitute the full 4:05 - 4:40 length uh protein that you wanted to deliver in the first place and this product looks exactly like the native uh protein. Now we"ve uh deployed this technology across the board across several therapeuti

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26/03/2026 10:21:03

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Mensaje de: jonhlucas70 Hora: 26/03/2026 10:16:29

This is how the pro drug product looks like. So you have two AV vectors uh the transine is split in in two halves each of them packaging half the transene and then when that is delivered at the protein level the product comes together and you have in this case the ABCA4 full length protein being made and restoring function in in these photo receptors. I will not go through uh the data in in of themology. I want to jump into the CNS data but one thing I wanted to show is that we have extensive expression data.

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26/03/2026 10:22:32

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Es mucho, resumido: SpliceBio es una compañía de terapias génicas centrada principalmente en oftalmología, pero que también está avanzando en sistema nervioso central (CNS). Su tecnología resuelve uno de los mayores problemas del sector: la limitación de tamaño de los vectores AAV. Para ello utilizan un sistema dual AAV que divide el gen en dos partes y lo reconstruye dentro de la célula mediante “protein splicing”, logrando una eficiencia comparable a un vector único. Su programa principal (LEAP) para la enfermedad de Stargardt ya ha completado la fase 1 sin problemas de seguridad y está entrando en fase 2 de eficacia con un ensayo controlado. Además, cuentan con validación en múltiples modelos (roedores y primates) y un pipeline con más de 20 targets, respaldado por una sólida propiedad intelectual y más de 200M$ levantados con inversores como Roche, Sanofi o Novartis.

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26/03/2026 10:22:47

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Mensaje de: jonhlucas70 Hora: 26/03/2026 10:22:32
Es mucho, resumido: SpliceBio es una compañía de terapias génicas centrada principalmente en oftalmo..........

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A nivel técnico, han demostrado que su tecnología funciona no solo en retina, sino también en cerebro, con resultados consistentes en primates, lo que amplía significativamente el potencial de mercado. Los datos muestran que el sistema dual AAV logra niveles de expresión y funcionalidad similares a los de un AAV tradicional, resolviendo una limitación clave de la terapia génica. En conjunto, SpliceBio está posicionada como una plataforma capaz de abordar enfermedades que antes no podían tratarse con terapia génica, con un pipeline activo en clínica y expansión hacia nuevas áreas terapéuticas.

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26/03/2026 10:24:48

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Mensaje de: JAVIFM Hora: 26/03/2026 7:01:14
Good morning

blablablablablabablabablabablabablabablabablabablababla

Rent. 3 Año: -47,20%

habelino

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26/03/2026 10:25:16

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Mensaje de: JAVIFM Hora: 26/03/2026 7:01:14
Good morning

pridicadores y bidentes

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26/03/2026 10:25:19

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Mensaje de: JAVIFM Hora: 26/03/2026 7:01:14
Good morning

Deberías empezar a medir la rentabilidad de tu tiempo empleado aquí

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26/03/2026 10:27:08

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Mensaje de: JAVIFM Hora: 26/03/2026 7:01:14
Good morning

que si 200M que si Roche que si pitos y flautas!!! jejejeje TRILEROS es poco! REALIDAD = facturación de ferretería y PERDIDAS Y emisión de PAPELAZO A 0,17!!! RESULTADOS MÁS DE LO MISMO Y FALTA DE CASH!!

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26/03/2026 10:28:18

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Mensaje de: JAVIFM Hora: 26/03/2026 7:01:14
Good morning