The Needle Issue #11


Welcome to the Needle, a newsletter from Haystack Science to help you navigate the latest translational research, with a roundup of the latest news on biotech startups from around the world.

This week we’re “stuck” on the fast-moving field of ‘molecular’ glue drugs. This is an area that is going places, with a steady stream of new ventures (by our count nearly 30 companies still in the preclinical stages), a burgeoning stream of papers describing new compounds in the literature and the first few programs, like Revolution Medicine’s daraxonrasib, moving through the clinic. Elsewhere, several government and venture initiatives promise to boost funding available to early-stage startups. Also, there is an interesting mix of financings and deals for startups developing advanced biologic approaches (rather than small molecules). Know of any that we may have missed? Please, let us know (info@haystacksci.com).

Haystack chat

Molecular glue degraders (MGDs) are currently having a bit of a moment. In the first half of 2025, the number of papers describing such compounds has doubled.

2025 has also witnessed a whole raft of MGD startups publish research related to their programs:

Startup Table
Startup (location) Scientific founders (location) 2025 paper
Ambagon Therapeutics (Eindhoven, The Netherlands) Michelle Arkin (UCSF, San Francisco, CA), Luc Brunsveld and Christian Ottman (Eindhoven University of Technology) Molecular glues of the regulatory ChREBP/14-3-3 complex protect beta cells from glucolipotoxicity
Cyrus Therapeutics (Seoul, South Korea) Keon Wook Kang (Seoul National University, Seoul, South Korea) High cereblon expression in neuroendocrine cancer confers vulnerability to GSPT1 molecular glue degrader
Matchpoint Therapeutics (Cambridge, MA) Nathanael Gray and Tinghu Zhang (Stanford University, Stanford, CA) and Edward Chouchani and Jianwei Che (Dana Farber, Boston, MA) Structure-guided design of a truncated heterobivalent chemical probe degrader of IRE1α
Monte Rosa Therapeutics (Boston, MA) Rajesh Chopra and Ian Collins (The Institute of Cancer Research and Cancer Research UK); Nicolas Thomä (Friedrich Miescher Institute, Basel, Switzerland) Structure-guided strategy for identifying human proteins predicted to be compatible with cereblon-based molecular glue degraders (see below for further details)
Oniria Therapeutics (Barcelona, Spain) Héctor G. Palmer, Esther Riambau, Isabel Puig, Josep Tabernero, Xavier Barril, and Carles Galdeano (Vall d’Hebron Institute of Oncology, University of Barcelona and ICREA) Cullin-RING ligase BioE3 reveals molecular-glue-induced neosubstrates and rewiring of the endogenous Cereblon ubiquitome
Proxygen (Vienna, Austria) Georg Winter (CeMM Research Center for Molecular Medicine, Vienna, Austria) Selective analysis of protein degradation by mass spectrometry enables degradome analysis and identification of direct protein substrates of molecular glues
Proteovant Therapeutics (King of Prussia, PA) Shaomeng Wang (University of Michigan, MI) Development of PVTX-405 as a potent and highly selective molecular glue degrader of IKZF2 for cancer immunotherapy
Sartar Therapeutics (Helsinki, Finland) Olli Kallioniemi and Harri Sihto (University of Helsinki, Finland) Pharmacokinetic profile and in vivo anticancer efficacy of anagrelide administered subcutaneously in rodents
SEED Therapeutics (King of Prussia, PA) Ning Zheng (University of Seattle, WA), Michele Pagano (New York University, NY) and Avram Hershko (Technion Institute of Technology, Haifa, Israel) UM171 glue co-opts CRL3 RING E3 ligase substrate coreceptor KBTBD4 as well as HDAC1/2, resulting in degradation of CoREST corepressors
Shenandoah Therapeutics (South San Francisco, CA) Jerry Crabtree and Nathanael Gray (Stanford University, Stanford, CA) A bivalent molecular glue linking lysine acetyltransferases to redirect p300 and CBP to activate programmed cell death genes normally repressed by the oncogenic driver, BCL6
Zenith Therapeutics (Basel, Switzerland) Daniel Nomura (UC Berkeley, CA); Nicolas Thomä (Friedrich Miescher Institute, Basel, Switzerland), and Martin Stahl (former Roche, LifeMine) Putative molecular glue niclosamide acts via ubiquitin E3 ligase CRL4AMBRA1-mediated degradation of cyclin D1 following mitochondrial membrane depolarization

On the commercial front, the march of startups receiving funding shows no sign of slowing down, with Trimtech Therapeutics and Booster Therapeutics raising substantive rounds. The first few months of the year have also seen the continuation of last year’s pharma MGD scramble to license programs from Triana Biomedicines and Neomorph, with deals based around molecular glues from Abbvie and Merck targeting Neomorph and Springworks, respectively.

In June, one of the flagship developers, Kymera Therapeutics, priced a $250.8 million follow-on offering (no mean feat in the present market) after announcing positive phase 1 safety data for KT-621, a novel MGD against STAT-6, and clinching a deal with Gilead Sciencesforanother small-molecule glue targeting cyclin-dependent kinase 2 (CDK2). All in all, we count 27 companies currently active in this preclinical space (Ambagon Therapeutics, Amphista Therapeutics, Booster Therapeutics, Captor Therapeutics, Cyrus Therapeutics, Degron Therapeutics, Dunad Therapeutics, F5 Therapeutics, Frontier Medicines, Lifemine Therapeutics, Magnet Biomedicine,Matchpoint Therapeutics, Montara Therapeutics, Monte Rosa Therapeutics, Neomorph, Oniria Therapeutics, Proxygen, Sartar Therapeutics, SEED Therapeutics, Shanghai Dage Biomedical Technology, Shenandoah Therapeutics, SK Biopharmaceuticals (Proteovant Therapeutics),Triana,Trimtech,Venquis Therapeutics, YDS Pharmatech, and Zenith Therapeutics). There are likely more.

Unlike their more recent cousins, the PROTACs (proteolysis targeting chimeras), MGDs have a long history. The archetypal MGD, thalidomide, was discovered back in the 1950s. From the late 1990s, a new generation of immunomodulatory imide drug (IMiD) derivatives of thalidomide were synthesized, culminating with the approvals of lenalidomide and pomalidomide for myeloma (which formed the basis for the Celgene (now BMS) franchise).

Unlike PROTACs, which use two ligands with a linker and tend to be rather unwieldy, MGDs are small, single compounds that induce conformational changes in E3 ubiquitin ligases and target proteins, reshaping both to enable binding. The vast majority of MGDs bind Cereblon (CRBN), leading to ubiquitination of the protein of interest and degradation in the 26S proteasome, although work is progressing to broaden MGD action to some of the other 600 or so E3 ubiquitin ligases (e.g., DCAF11,15 or 16, DDB1, SIAH, KEAP1, VHL, β-TrCP, Nedd1 and, just last week, TRIM21).

A key challenge in finding new MGDs has been a lack of understanding of the structural rules whereby MGDs turn their target proteins into CRBN ‘neosubstrates’, which has meant MGD ‘hit-finding’ is much more challenging, with fewer degrees of freedom than PROTACs.

What drug hunters have established is that many protein targets of glues contain a β-hairpin structural motif known as the ‘G-loop’. When a MGD brings a target together with CRBN, one end of the MGD interacts with a binding pocket in the C-terminal domain of CRBN, while the other end protrudes from the pocket and interacts with the G-loop (part of the so-called ‘degron’) in the neosubstrate. But how many proteins possess the β-hairpin G-loop or whether the loop is strictly necessary for MGD action have remained open questions. A recent study by Monte Rosa Therapeutics’ scientists starts to tackle these issues, disclosing a large cadre of potential new substrates for CRBN, some of which depart from the canonical β-hairpin G-loop, radically expanding MGD target space.

To map the full range of proteins potentially recruitable by CRBN through MGDs, the team led by John Castle and Sharon Townson developed computational algorithms to search for β-hairpin G-loop motifs in protein structures from two databases: Protein Data Bank and AlphaFold2. This approach resulted in 1424 candidate proteins, some of which were experimentally validated in MGD assays. The list included previously known neosubstrates, but also new proteins such as NEK7—a protein of interest as an autoimmunity target.

The researchers then wondered if the full β-hairpin structure of the G-loop is required for CRBN recognition and rescreened the structure databases looking for a minimal, structurally defined helical G-loop motif. This resulted in the identification of 184 additional potential neosubstrates, including mTOR, a well-established therapeutic target for drugs like rapamycin and sirolimus. Crystallographic data showed that the binding of this helical G-loop to CRBN is similar to that of the canonical β-hairpin G-loops.

As these protein–protein interactions have been well characterized, the team then tried to identify an even wider set of potential neosubstrates, looking now for proteins with sequences that might result in surfaces with electrostatic properties similar to known CRBN interactors, independently of secondary structure and the existence of G-loops. Using surface-matching algorithms, they identified and validated VAV1 (another autoimmune disease target) as a CRBN neosubstrate, providing compelling evidence that G-loops are not strictly necessary for the action of MGDs.

These findings show that CRBN recruitment through MGDs can be driven by a broader set of structural features than previously thought. The identification of a large number of neosubstrates potentially opens up a whole new set of previously ‘undruggable’ targets to MGDs (>1,600 proteins from many target classes, according to the Monte Rosa team).

The big questions, though, are still ahead. How will drug developers mitigate the risks of ‘off-tissue’ toxicity as this swathe of novel MGD compounds and new targets make their way into the clinic?One answer to the toxicity concern is molecular glue antibody conjugates (MACs), which can better localize glues to the tissue of interest. But that’s a subject for a whole other future Haystack Chat!

Papers: Best of the rest

Target biology

Neuronal HDAC3 knockdown promotes propriospinal detour pathway formation and locomotor recovery in a mouse model of spinal cord injury | Science Translational Medicine

NEDD4L induces mitochondrial dysfunction and neurodegeneration by promoting LIPT2 degradation in Huntington’s disease | PNAS

An alternate receptor for adeno-associated viruses | Cell

Disruption of the KLHL37–N-Myc complex restores N-Myc degradation and arrests neuroblastoma growth in mouse models | JCI

Sensory neuron–expressed FGF13 controls nociceptive signaling in diabetic neuropathy models | JCI

Microbially produced imidazole propionate is a driver of atherosclerosis in mice and is elevated in human plasma from atherosclerosis patients | Nature

CRISPR-Cas9 screening in cell lines and mouse xenograft models reveals DP-diacylglycerol synthase 1 (CDS1)/CDS2 axis as key target in uveal melanoma ǀ Nature Genetics

Polyglycine-mediated aggregation of FAM98B disrupts tRNA processing in GGC repeat disorders | Science

Platforms

Computational structure-based discovery of positive allosteric modulators of the A1 adenosine receptor | PNAS

Tuning antibody stability and function by rational design of framework mutations ǀ mAbs

CRISPR-Cas9–edited human CD34+ cord blood cells transplanted into mice with targeted immune system gene knockouts as platform for generating humanized models of blood cell-derived disorders ǀ Science Advances

Tool and lead compounds

An orally available Mpro/TMPRSS2 bispecific inhibitor with potent anti-coronavirus efficacy in vivo | Nat Comms

Molecular glues targeting TRIM21 (TRIMTACs) degrade protein aggregates in cells ǀ Nat Comms

Biologics

First-generation and preclinical evaluation of an EphA5-targeted antibody-drug conjugate in solid tumors | JCI

Human frataxin fused via a diglycine linker to the cell-penetrant Tat peptide (nomlabofusp) delivers functional frataxin into mitochondria in vitro and restores frataxin in buccal swab tissue samples taken from phase 1 study subjects ǀ AAPS Journal

Personalized or rare-disease therapies

Microglia replacement halts the progression of microgliopathy in mice and humans | Science

Coenzyme Q headgroup intermediates can ameliorate a mitochondrial encephalopathy | Nature

A multi-adjuvant personal neoantigen vaccine generates potent immunity in melanoma | Cell

n-Lorem splice-modulating 18-mer phosphorothioate 2' methoxyethyl ASOs target a pathogenic intronic variant in adult polyglucosan body disease to correct mis-splicing and restore enzyme activity in patient cells ǀ Nucleic Acids Res

Delivery approaches

Systemic administration of an RNA binding and cell-penetrating antibody targets therapeutic RNA to multiple mouse models of cancer | Science Translational Medicine

Oral delivery of liquid mRNA therapeutics by an engineered capsule for treatment of preclinical intestinal disease | Science Translational Medicine

Controlled colonization of the human gut with a genetically engineered microbial therapeutic | Science

Advanced cell and gene therapies

CAR-T cells engineered to secrete killer cell engagers (BIKEs) recruit natural killer cell specificity to potentiate antitumour responses in cell lines/mouse models of Burkitt’s lymphoma cells and ovarian cancer | Nature Biomedical Engineering

Potentiating checkpoint blockade: Bispecific nanobody engagers of CTLA-4/PD-L1 and circulating host polyclonal immunoglobulins enhance ADCC or CDC in mouse models of melanoma and colon cancer| Nature Biomedical Engineering

REGENEXBIO’s microdystrophin containing an extended C-terminal domain shows higher muscle transduction, improved muscle force and resistance to damage in mdx mice ǀ Mol Ther Methods Clin Dev

Preclinical goings on

Overview of the financing and deals outlook for biotech (warning: not easy reading!):

Endpoints: Biotech’s Q2 numbers paint a picture of the good, the bad and the ugly

HSBC analysis of venture financing in the first half of 2025: “headwinds and uncertainty lingering”

But there were several promising announcements for those in search of funding: Several initiatives proposing funding for early-stage biotech startups and translational investigators:

The University of Tokyo Edge Capital Partners closes a new $326 million fund for startups

$100 million North America University Innovations Fund co-launched by Longview Innovation and Anzu Partners to invest in technologies out of US national labs and universities

UK government launches 10-year plan to galvanize the country’s life sciences sector.

Insightful Nucleate analysis of where VC funding is by region

In accelerator news:

BioMed X establishes new XSeed Labs incubator site at Servier’s research and development site in Paris-Saclay

Also some interesting initiatives to galvanize treatments for rare diseases that have been traditionally unattractive for commercial development:

Chan Zuckerberg Initiative (CZI) provides $20 million for UCSF to launch new Center for Pediatric CRISPR Cures

UK Rare Disease Research Platform established with £14 million investment over five years

Elpida Therapeutics' innovative business model for developing a treatment for an ultrarare disease

The ‘dialog’ between US research universities and the Trump administration continues:

Coalition of 200 US academic organizations proposes alternatives to the Trump administration’s plan to cut billions of dollars in research overhead/indirect costs

And finally, we grab a Pimm’s and congratulate all those new ventures receiving accolades and recognition during the 2025 award season this summer:

Life Sciences British Columbia 2025 startup awards highlight NanoVation Therapeutics, Variational AI, CereCura Nanotherapeutics and Optigo Biotherapeutics

Myricx Bio, a startup developing antibody-drug conjugates with small-molecule N-myristoyltransferase inhibitors as payloads for solid tumors, received “The UK Cancer Research Horizons Start-Up Achievement Recognition Award for 2025”

UK’s Enterprise Investment Scheme Association Awards 2025 commends Exonate, a Cambridge-based startup developing eye drop treatments for diabetic eye disease and wet age-related macular degeneration

Preclinical financings

Biotech Funding Table
Date Company (location) Amount (millions) Funding type (lead investors) Therapeutic (lead) focus
July 2, 2025 Azure Cell Therapies (Lausanne, Switzerland) CHF 0.65 Grants (InnoSuisse) Large-scale manufacture of allogeneic mesencephalic dopaminergic progenitors for Parkinson’s disease
July 8, 2025 Ciloa (Montpellier, France) €6.5 Grant (France 2030 ‘Biotherapies and Biomanufacturing of Innovative Therapies’) Recombinant adiponectin packaged in stable extracellular vesicles produced by human cell lines for type 2 diabetes and obesity
July 8, 2025 Centivax (South San Francisco, CA) $45 Series A (Future Ventures) Universal influenza vaccine comprising an undisclosed lipid nanoparticle delivering a cocktail of 21 different influenza hemagglutinin mRNAs and an mRNA for an undisclosed influenza neuraminidase or nucleoprotein
July 9, 2025 Actithera (Oslo, Norway) $75.5 Series A (M Ventures, Hadean Ventures, Sofinnova Partners, and 4BIO Capital) ¹⁷⁷Lu or ²²⁵Ac chelators bound via undisclosed linker chemistry to small-molecule covalent binders of fibroblast-activation protein for solid tumors
July 10, 2025 Renasant Bio (Berkeley, CA) $54.5 Seed (5AM Ventures) Oral small-molecule correctors/chaperones that restore proper folding/trafficking and potentiators that restore channel function in mutated polycystin 1 (PC1)/PC2 for autosomal dominant polycystic kidney disease
July 14, 2025 Oncomatryx (Bilbao, Spain) €2.5 Grant (European Innovation Council Accelerator) Antibody-drug conjugates against fibroblast activation protein and other targets on cancer-associated fibroblasts in tumor microenvironment for immune-cold metastatic pancreatic, MSS colorectal and non-small cell lung cancer

Preclinical deals

Biotech Deal Table
Date Type Payer (location) Payee (location) Upfront payment (millions) Milestones amount (millions) Total (up to millions) Therapeutic Focus
July 8, 2025 Strategic partnership Adagene (Suzhou, China) ConjugateBio (Princeton, NJ) ND ND ND Undisclosed bispecific antibody-drug conjugate with pH-sensitive PEG8- and triazole-containing p-aminobenzyloxycarbonyl-peptide-maleimidocaproyl linker to camptothecin for solid tumors
July 10, 2025 Acquisition Cresset (Cambridge, UK) Molab.ai (Munich, Germany) ND ND ND Virtual library screens with ML ADME prediction and generative models for chemotype design
July 10, 2025 Licensing AbbVie (North Chicago, IL) IGI Therapeutics (New York, NY) $700 $525 $1,225 Trispecific T-cell engager that targets BCMA and CD38 on myeloma cells and CD3 on T cells for relapsed/refractory multiple myeloma
July 10, 2025 Strategic partnership Secarna Pharmaceuticals (Martinsread, Germany) Vect-Horus (Marseille, France) ND ND ND Secarna’s locked nucleic acid (LNA) oligonucleotide therapeutics to combine with Vect-Horus’ VHH antibody vectors targeting transferrin receptor 1 to cross the blood-brain barrier for CNS diseases
July 17, 2025 Strategic partnership NKGen Biotech (Santa Ana, CA) HekaBio (Chuo-ku, Japan) ND ND ND HekaBio to lead regulatory, manufacturing, and commercial development of NKGen’s ex vivo expanded autologous NK cells for Alzheimer's and Parkinson’s diseases
July 17, 2025 Acquisition I-Mab (Rockville, MD) Bridge Health Biotech (New York, NY) $1.8 $2.075 $3.875 Bispecific and multi-specific formats of antibodies and antibody-drug conjugates based on high-affinity parental mAb targeting claudin 18.2

Stay in touch

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If you’re interested in commercializing your science, get in touch. We can help you figure out the next steps for your startup’s translational research program and connect you with the right investor. Follow us on X, BlueSky and LinkedIn. Please send feedback; we’d love to hear from you (info@haystacksci.com).

Until next time,

Juan Carlos and Andy

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