FreeBioAlpha
@freebioalpha
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Open sourcing information to activate more talent in science. Powered by @axialxyz
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Hello world 🌎
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Scientist Stories: Viviana Gradinaru, Machine Learning Assisted Directed Evolution
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Early-Stage Biotech Value Creation: The Roles of Equity and Partnerships
Find more resources to build bio companies at FBA
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How are DNA-encoded libraries created?
DNA-encoded libraries (DELs) are created by covalently attaching a unique DNA barcode to each compound in a library. Which is then incubated with the target protein, and the bound compounds are eluted and amplified.
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Founder Stories: Arie Belldegrun, Founder of Kite Pharma
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How are non-canonical amino acids produced?
Non-canonical amino acids are amino acids that are not encoded by the standard genetic code. And can be produced with genetic, enzymatic, and chemical methods:
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Omaveloxolone
An oral Nrf2 activator approved to treat Friedreich's ataxia from modification of oleanoic acid
Developed by Reata and acquired by Biogen
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PipeBio
@pipe_bio
is a cloud-based bioinformatics platform for biologics discovery. It allows wet lab scientists to easily analyze antibody and peptide sequences with functional assay data, and bioinformaticians to deploy their own code and run workflows
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Eclipsebio
@bio_eclipse
:
- Founded in 2017 to develop RNA genomics and epigenetics products
- RBP-eCLIP to recognize sequence/structure motifs
- m6A-eCLIP to profile m6A sites
- miR-eCLIP to map miRNA/mRNA interactions
- End-seq to map 5' and 3' ends of RNA transcripts
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Worth a Read: 'Unexpected Effects of Protein Degradation'
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Pixelgen Technologies
@PixelgenTech
, founded in 2020 by Simon Fredriksson (former CEO/co-founder of Olink), develops spatial proteomics products. Mapping cell surface proteins and their spatial inter-relationships.
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PF-07054894
An oral, selective CCR6 antagonist in Phase I clinical trials for ulcerative colitis
Identified from directed screening for novel negative allosteric modulators by Pfizer
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Fenebrutinib
An oral BTK inhibitor that recently demonstrated efficacy in multiple sclerosis
Developed by Genentech and is now entering phase III trials
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How do integrin proteins work?
Integrins are a family of cell surface receptors that mediate the attachment of cells to the extracellular matrix (ECM). They are heterodimers, consisting of an α subunit and a β subunit.
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Orforglipron
A non-peptide GLP-1R agonist in Phase III clinical trials for obesity & type 2 diabetes
Identified through high-throughput screening, with origins from Chugai and now developed Eli Lilly
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Scientist Stories: Frances Arnold, Directed Evolution and Protein Engineering
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AZD4747
A brain-penetrant KRASG12C inhibitor lead
To treat KRASG12C-positive tumors from structure-based drug design by AstraZeneca
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Expansion Therapeutics
@expansionrx
:
- Founded in 2016 to develop small molecules targeting RNA
- ID'ing druggable RNA structures
- Building a pipeline to treat myotonic dystrophy type 1 (targeting splicing), ALS & FTD (translation), and tauopathies
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Atrasentan
An oral ETA receptor antagonist in Phase III clinical trials for IgA nephropathy & Phase II trials for Alport Syndrome, designed based on prior non-peptide ETA receptor antagonists from Abbott Labs
Developed by Chinook Therapeutics (acquired by Novartis)
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High-Throughput Chemistry and Engineering with Kadi Saar
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Scientist Stories: Seema Lakdawala, Assembly of Viruses
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Think Bioscience
@ThinkBioscience
is a synthetic biology company developing small molecule therapeutics against difficult-to-drug proteins. Founded in 2019 out of Boulder, Colorado, the company develops microbial systems to evolve targeted and readily synthesizable drug leads.
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Ascidian Therapeutics
@AscidianTx
:
- Founded in 2020 to develop exon-editing RNA drugs
- With its platform replacing mutated exons at the RNA level
- Targeting genes with high mutational variance while maintaining native gene expression
- First focusing on ABCA4 retinopathy
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Attovia Therapeutics:
- Founded in 2022 as a spinout from Alamar Bio
- To use their biparatopic nanobody platform, Attobody, to generate binders with accelerated internalization & increased tissue penetration
- Focusing on immune-mediated disease
- Raised $60M led by Frazier
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The Entrepreneur's Guide to a Biotech Startup
Find more resources to build bio companies at FBA
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ProQR Therapeutics
@ProQR
:
- Founded in 2012 to develop RNA editing medicines
- Centered around their Axiomer (ADAR) platform
- Licensing IP from Radboud & Leiden University Medical Centers
- Developing candidates for NTCP, NASH, among others and collaborating with Eli Lilly
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Delix Therapeutics
@Delix_tx
:
- Founded in 2019 to develop non-hallucinogenic psychedelics
- To modify neuroplasticity to develop therapies for treatment-resistant depression, substance use disorder, PTSD, cognitive impairment in schizophrenia, and neurodegeneration
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Nimbus Therapeutics
@NimbusTx
raised $210M co-led by GV, SR One, and Atlas Venture
For its 3rd chapter, now developing programs for Werner syndrome, an undisclosed autoimmune target, and NDI 1150-101 to target HPK1 as a cancer treatment
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Danaher buys Abcam for $5.7B to bring on the latter's product portfolio of antibodies, reagents, biomarkers and assays. Reportedly outbidding Agilent during the M&A process that evaluation ~20 bidders.
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Camlipixant
A phase III oral, twice-daily P2X3 receptor antagonist for first-line treatment of refractory chronic cough
Developed by AstraZeneca, Neomed, Bellus Health & GSK
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Transforming biomanufacturing using software with Martin Permin
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FLX475
An oral CCR4 antagonist in Phase II trials for immunooncology
Discovered through optimization of prior CCR4 ligands from RAPT Therapeutics
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How do proteins cross the plasma membrane?
1. Passive diffusion: Small, hydrophobic proteins can diffuse directly through the phospholipid bilayer of the plasma membrane. However, this process is very slow and only works for very small proteins.
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Scientist Stories: William Schief, Structure Assisted Vaccine Discovery for Infectious Diseases
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ADARx:
- Raised $200M co-led by Bain Capital & TCG Crossover
- To use its RNA targeting platform (RNAi and editing) for genetic, cardiometabolic & CNS diseases
- With its lead asset, ADX-324, in development for hereditary angioedema with ADX-038 for complement mediated diseases
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Founder Stories: Joshua Boger, Founder and CEO of Vertex Pharmaceuticals
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What is a protein switch pocket?
A protein switch pocket is a small region of a protein that can undergo a conformational change in response to a ligand binding. This conformational change can alter the protein's activity, function, or localization.
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Decibel Therapeutics was acquired by Regeneron for $109M
With an additional non-tradeable contingent value right (CVR) to receive up to $104M upon achievement of certain DB-OTO development and regulatory milestones
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How are chimeric antigen receptors created?
Chimeric antigen receptors (CARs) are created by fusing three different components: antigen-binding, transmembrane , and intracellular signaling domains
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SpyGlass Ophthalmics:
- Founded in 2019 to develop ophthalmic therapeutic devices
- Raised $90M led by RA Capital
- To conduct multiple US clinical trials for their delivery platform in glaucoma and other chronic ophthalmic diseases
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Alltrna
@alltrna
:
- Founded in 2018 to develop programmable tRNA medicines
- Delivering tRNAs to restore disrupted protein production and treat diseases with the same underlying genetic mutation
- Mapping out the universe of tRNAs and their effects on translation/human disease
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Scientist Stories: Aly Khan, Computational Approaches to Study Immune Cells
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FreeBioAlpha
Open sourcing information to activate more talent in life sciences
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Worth a Read: 'Unexpected Effects of Protein Degradation'
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Rapport Therapeutics raised $150M led by Cormorant Asset Management. To precision medicines for neurological disorders. Founded in 2022 based on work from David Bredt while he was at Janssen Neuroscience, Rapport focuses on receptor-associated proteins (RAPs).
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Scientist Stories: Jennifer Lewis, Printing Synthetic and Living Matter in Three Dimensions
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Vorasidenib
An oral first-in-class dual IDH1/IDH2 inhibitor with efficacy in Phase III trials for IDH-mutant grade 2 gliomas, discovered through structure-based drug design (SBDD) of a prior mIDH inhibitor, developed by Agios and Servier
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Neumora
@NeumoraTx
filed their S-1 to go public. After raising more than $600M to build a pipeline of precision medicines for brain diseases
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What are Yamanaka factors?
Yamanaka factors are a group of 4 transcription factors that can be used to reprogram adult cells into induced pluripotent stem cells (iPSCs). That have the potential to develop into any type of cell in the body:
1. Oct4
2. Sox2
3. Klf4
4. c-Myc
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Scientist Stories: Cynthia Kenyon, Staying Ahead of the Curve
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Gepotidacin
An oral, twice-daily antibacterial Type II topoisomerase inhibitor for the treatment of uncomplicated UTIs
In phase III trials developed by GSK
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Shape Therapeutics
@ShapeTx
:
- Founded in 2018 to develop RNA-targeting medicines to repair the genetic causes of disease
- Combining ADAR technology with next-generation AAVs to develop drugs for rare disorders to Alzheimer’s, Parkinson’s, and more
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Kate Therapeutics:
- Founded in 2020 to develop AAV gene therapies to treat genetically defined muscle & heart diseases
- Raised $51M co-led by Versant & Westlake Village BioPartners
- A pipeline focused on DM1 & FSHD
- Also announcing a partnership with Astellas to treat XLMTM
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Scientist Stories: Jim Allison, Pioneering Immunotherapies
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Worth a Read: 'Large Language Models in Molecular Biology'
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Acoramidis
An oral TTR stabilizer in phase III trials in ATTR-CM
ID'ed from a HTS for TTR ligands and developed by BridgeBio
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Founder Stories: Laura Walker, Generation of Neutralizing Antibodies for Pandemics
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Worth a Read: 'Biotech Models: Virtual Reality vs Virtual Mythology'
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Brigimadlin
An oral MDM2–p53 PPI inhibitor in phase II/III trials for DDLPS
Designed from prior spirooxindoles by Boehringer Ingelheim
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RMC-6291
An oral KRASG12C(ON) tri-complex inhibitor for the treatment of advanced solid tumors
In phase I/Ib trials developed by Revolution Medicines
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Septerna
@Septerna_Inc
:
- Founded in 2019 to discover small molecule drugs against GPCRs
- Raised $150M led by RA Capital
- To develop is pipeline of candidates focusing on its lead program targeting the parathyroid hormone 1 receptor (PTH1R) to the clinic
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How do nanopores work?
Nanopores are tiny holes that can be used to measure the electrical properties of molecules as they pass through. This makes them useful for DNA sequencing and protein analysis
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How does phage-assisted continuous evolution work?
Phage-assisted continuous evolution (PACE) is a technique for evolving proteins by using bacteriophages to amplify and select for advantageous mutations
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Superluminal Medicines raised $33M led by RA Capital. To build a generative chemistry platform, initially focused on small molecules for high-value G protein-coupled receptor (GPCR) targets. Working to generate candidate-ready compounds with differentiated TPPs in months.
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Scientist Stories: Magdalena Zernicka-Goetz, Human Development and Synthetic Embryos
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Uncommon
@uncommonbio
:
- Founded in 2018 by Benjamina Bollag to develop cultivated pork
- Raised $30M led by Lowercarbon Capital
- Using iPSCs to differentiate into multiple cell types such as muscle and fat
- Focusing on bacon & pork belly with plant-based & cell-cultured meat
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CT3
An irreversible trypanosomal topoisomerase II inhibitor
Showing preclinical efficacy in mouse models of Chagas disease and sleep sickness
Discovered through a phenotypic screen against trypanosomes by Novartis (NITD)
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Myeloid Therapeutics
@MyeloidTx
:
- Founded in 2019 by Daniel Getts, Ron Vale, Siddhartha Mukherjee, Tom Cahill, and Yuxiao Wang
- Raised $73M led by Hatteras with ARCH, 8VC, and Newpath
- Using mRNA to reprogram cells in vivo
- With a lead in phase 1/2 trials for T-cell lymphoma
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Glyphic Biotechnologies
@GlyphicBio
, founded in 2021, is building a protein sequencing platform to discover & measure proteoforms. Which are a specific molecular form of a protein encoded by gene. Arising from SNPs/CNVs, alternative splicing, and/or PTMs
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Founder Stories: Kiran Mazumdar-Shaw, Founder & Chairman of Biocon
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Oova Fertility
@OOVAlife
:
- Founded in 2017 by Aparna Divaraniya
- Raised $10.3M led by Spero Ventures
- To commercialize a personalized fertility diagnostic device
- Measuring hormones present in women’s urine through a paper-based strip for cycle & hormone pattern management
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AAVantgarde Bio:
- Founded in 2021 out of Italy by Alberto Auricchio
- Raised €61M co-led by Forbion & Atlas Venture
- Developing AAV gene therapies for inherited retinal disorders
- With 2 candidates: Usher1B retinitis pigmentosa, using dual hybrid & Stargardt disease, intein
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Moderna announced a new partnership with Immatic to develop cancer vaccines & cell therapies, in a deal worth up to $1.7B. With $120M paid upfront
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Enveda Biosciences
@envedabio
is a drug discovery company, founded in 2019, that uses machine learning, metabolomics, and robotics to discover new medicines from nature's chemistry. Developing small molecules for complex diseases.
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Korro Bio
@KorroBio
:
- Founded in 2018 to develop RNA editing therapies
- Raised $117M & going public through a reverse merger with Frequency
- To use its ADAR platform focusing on a lead program in alpha-1 antitrypsin deficiency & getting to the clinic in 2024
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CFT1946
An oral, mutant-selective BRAFV600X degrader
In phase 1 trials to treat BRAF-driven cancers developed C4 Therapeutics
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Locanabio
@locanabio
:
- Founded in 2016 to develop RNA-targeting drugs
- Focusing on neurodegenerative, neuromuscular & retinal diseases
- With its CORRECTX platform relying on ADAR systems that modify RNA cleavage, splicing, gene replacement, translation enhancement & editing
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Amber Bio:
- Raised $26M co-led by Playground and a16z Bio
- To develop their RNA editing platform for multi-kilobase edits, allowing a single drug to treat diseases with high allelic diversity
- Centered around inventing drugs through biologically inspired design
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JT001
An oral NLRP3 inflammasome inhibitor
Discovered from oral efficacy models and related to a phase I candidate GDC-2394 from MCC950 & optimized for lipophilic ligand efficacy
Developed by Jecure and led by Genentech
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Scientist Stories: Mark Davis, Exploring Human Immunology
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Eighth Day of Creation
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Worth a Read: 'Discovery moments: TYK2 psuedokinase inhibitor'
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hC Bioscience:
- Founded in 2021 to develop tRNA-based therapeutics
- With its PTCX (Patch) platform using engineered tRNAs to recognize nonsense mutations in mRNA
- Suppressing them by inserting an amino acid where it is needed, restoring the protein to full length and function
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How does RNA fold?
RNA folds in a hierarchical manner, with the formation of secondary structure occurring first, followed by tertiary structure
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Aledade
@AledadeACO
:
- Founded in 2014 by Farzad Mostashari & Matthew Kendall
- To help physicians form/operate accountable care organizations
- Raised $260M led by Lightspeed
- Scaling its360° practice support, workflow optimization, ACO governance & support services
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Scientist Stories: Li-Huei Tsai, Molecular, cellular, & circuit basis of neurodegenerative disorders
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Strive Health
@StriveHealthUS
:
- Founded in 2018 to build value-based healthcare products for kidney disease
- Raising $166M led by NEA
- Serving >80K CKD and ESRD patients across 30 states
- Providing management & support as well as dialysis and kidney transplants
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How is protein structure determined by cryo-EM?
Cryo-electron microscopy (cryo-EM) is a technique used to determine the three-dimensional structure of proteins and other biological molecules
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Alkeus Pharmaceuticals
@alkeuspharma
:
- Founded in 2011 by Leonide Saad & Ilyas Washington to develop drugs for eye diseases
- Raised $150M led by Bain Capital
- Supporting the registration & launch of gildeuretinol (ALK-001), a potential treatment of Stargardt disease
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FogPharma
@FogPharma
:
- Founded in 2015 to develop cancer medicines against 'undruggable' targets
- Using their stapled peptide technology
- Their Helicon polypeptide-based products are designed to cause the transmembrane & target cancer-causing proteins
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